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Cure SMA

If someone you know has been recently diagnosed with SMA, please contact infopack@curesma.org to request a packet of information on SMA, as well as other resources available to families.

 

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© Cure SMA

Dear Friend,

Cure SMA is focused on advancing our role as the leader in spinal muscular atrophy (SMA) by expanding our transformative research and care programs. With multiple treatments approved by the U.S. Food and Drug Administration (FDA), symptom and muscle-targeting treatments in development, and combination therapies on the horizon, it is imperative that we increase access to excellent SMA care options for all people living with SMA.

The Cure SMA Care Center Network and SMA Clinical Data Registry is expanding our local presence, ensuring individuals with SMA can seek treatment and experience the best possible outcomes, and improving our understanding of the effects of SMA. Together, this will help us establish an evidence-based standard of care and shape a stronger future for all individuals with SMA and their families.

You may be asking yourself, what can I do to support the SMA community and help expand access to the best possible treatment and care options?

One the easiest and most effective ways that you can help is to make a gift to Cure SMA today. We are uniquely positioned to make the greatest impact for the SMA community. Beginning today, and running from November 17-19, anyone who donates $50 or more will be entered for a chance to win two (2) Cure SMA Face Masks. Please donate and tell your friends and family about this exciting opportunity!

Thank you in advance for your support. 

Very Best,

Erin Kelly
Vice President, Development 

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

Although this year has brought with it many challenges, the SMA community continues to demonstrate its strength and resilience. Cure SMA remains committed to bringing you the information, resources, and support you need as we continue to navigate the COVID-19 pandemic.

Last week, the Advisory Committee on Immunization Practices (ACIP)—a U.S. Centers for Disease Control and Prevention (CDC) committee of healthcare professionals providing guidance on the use of vaccines—recommended Phase 1a of its proposed phased approach for the allocation of vaccines for COVID-19, starting with healthcare workers and residents at long-term care facilities.

We know that many of you are concerned about vaccine allocation once a vaccination is available. Cure SMA continues to push for priority access to COVID-19 vaccines for the SMA community, advocating directly to the committee and as part of larger healthcare and disability coalitions. ACIP is expected to consider other phases of its allocation framework once COVID-19 vaccines are approved and released.

The ACIP recommendations are a framework that will guide decisions at the state and local level. State and local public health officials will determine distribution of the COVID-19 vaccine in their communities, which is why Cure SMA has also focused our advocacy in states, educating Governors early on in their planning process about the needs of the SMA community. Finally, we developed a resource for the SMA community to use to self-advocate for access to COVID-19 vaccine at the provider level.

ACIP’s initial recommendations were released in advance of the U.S. Food and Drug Administration (FDA) meeting this on December 10. On Thursday, the FDA will discuss the Emergency Use Authorization of the Pfizer-BioNTech vaccine candidate for the prevention of COVID-19 in individuals 16 years of age and older. This is one of the final steps toward approval for the vaccine, at which time the companies will begin the distribution process. Biotechnology company Moderna is also expected to see a review of its vaccine candidate in the coming weeks after a recent filing with the FDA for emergency authorization.

The good news is that vaccines for COVID-19 are on the horizon, and Cure SMA believes that vaccination will be the best defense against this virus. However, there are still a lot of questions to be answered related to the efficacy and safety of available vaccines, who will be eligible to receive the vaccines (i.e., age of recipients), as well as plans around vaccine access. Cure SMA will be closely tracking the available data from vaccine clinical trials and continues to educate and engage with decision-makers to ensure they consider the perspectives and experiences of the SMA community in their recommendations for vaccine distribution.

As we learn more, we will be sharing any FAQs and new resources about approved COVID-19 vaccines on the Cure SMA COVID-19 Information Center—keep an eye out for updates. We also anticipate hosting our next COVID-19 Community Webinar in early 2021, sharing more perspectives and recommendation on COVID-19 vaccination as it specifically relates to the SMA community.

Finally, while the current progress with vaccines for COVID-19 looks to be very positive, Cure SMA still strongly recommends that the SMA community continue the highest levels of isolation, given we are in the middle of the winter virus season and at the highest peak so far in this pandemic.

Sincerely,

Kenneth Hobby,
President, Cure SMA

Dr. Mary Schroth
Chief Medical Officer, Cure SMA

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Dear Friend,

For the past two years, Cure SMA’s Summit of Strength program has brought together thousands of SMA community members for the opportunity to network and learn about the latest advances in treatment, care, advocacy, and support. In the midst of the COVID-19 pandemic, we''re grateful to be able to continue these opportunities through the Summit of Strength Virtual Webinar Series. 

Upcoming Summit of Strength Webinars include:

May 20th Physical Therapy at Home

• Tina Duong, MPT, PHDc, Physical Therapist and Clinical Research Manager with the Division of Neuromuscular Medicine at Stanford University
• Representative from Biogen

REGISTER ➱

May 27th SMA Nutrition: Now More Than Ever

• Stacey Tarrant, RD, LDN, Clinical Nutrition Specialist II at Boston Children’s Hospital
• Representative from Genentech

REGISTER ➱

Keep an eye on Cure SMA’s site for added webinar topics. We hope you will register for these webinars and submit questions in advance to help drive the conversation!

If you have any questions about the Virtual Summit of Strength Webinars, please contact familysupport@curesma.org.

Thank you to the National Presenting Sponsors for the 2020 Summit of Strength Program: AveXis, Biogen, and Genentech. We would also like to recognize Platinum Sponsor, Scholar Rock, for its support.

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

This is a reminder to register for Cure SMA''s second COVID-19 SMA Community Webinar on Thursday, May 28th, at 1:00 p.m. ET/12:00 p.m. CT/11:00 a.m. MT/10:00 a.m. PT. You will hear from the Cure SMA team including Kenneth Hobby, President of Cure SMA; Mary Schroth, M.D., Chief Medical Officer; Colleen McCarthy O''Toole, VP of Events & Family Support; and Maynard Friesz, VP of Policy & Advocacy.

REGISTER ➱

The agenda for the 60-minute webinar will be shaped by your questions submitted in advance. Please submit your questions when you register or by emailing webinar@curesma.org by end of day Thursday, May 21st. We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

To watch Cure SMA''s previous COVID-19 SMA Community Webinar from March 27th, please click here.  

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

To the full spinal muscular atrophy (SMA) community, we want you to know that Cure SMA is continuing to support you with information and resources related to the ongoing COVID-19 pandemic. The following frequently asked questions (FAQs) are designed to help address key concerns expressed by members of the SMA community about COVID-19 vaccination and provide additional information about how you can protect yourself and your family. We encourage you to refer to the Centers for Disease Control and Prevention (CDC) for the most up-to-date information, and we will update our FAQ as often as needed.

Read all Cure SMA communications and guidance to date related to the pandemic by visiting the Cure SMA Coronavirus (COVID-19) Information Center. If you have any questions, please email info@curesma.org.

Frequently Asked Questions 

Last updated December 13, 2020

When do we expect a COVID-19 vaccine to be available in the U.S.?
The first COVID-19 vaccine was approved by the U.S. Food and Drug Administration (FDA) for Emergency Use Authorization on December 11, 2020, and shipment of the vaccine to states is expected to be immediate. A second COVID-19 vaccine candidate will be reviewed by the FDA on December 17, 2020. Learn more about the latest on vaccines tracking here.

When do you believe people with SMA will be eligible to get a COVID-19 vaccine?
Approved COVID-19 vaccines will be made available to U.S. adults in phases. A federal vaccine advisory committee proposed a phased approach and recommended starting with healthcare workers and residents at long-term care facilities in Phase 1a. The committee is proposing adults with high-risk medical conditions be included in Phase 1c. However, the committee has not finalized its Phase 1c recommendations, including timeline and who will be considered as being high-risk. Cure SMA continues to push for priority access to COVID-19 vaccines for the SMA community, advocating directly to the committee and as part of larger healthcare and disability coalitions.

Will the COVID-19 vaccines be available and safe for my child with SMA?
Based on available clinical data, we expect the approved COVID-19 vaccines to be made available primarily to adults, either 16 or 18 years of age or older, depending on the vaccine. However, clinical trials continue to expand those recruited to participate, and therefore the groups recommended to receive the vaccines could change in the future. As of now, a COVID-19 vaccine will not be available for young children until more studies are completed.

Should I take a COVID-19 vaccine once it is available to me or my child?
Cure SMA believes that vaccination will be the best defense against the COVID-19 virus. When available to people with SMA, we recommend speaking with your healthcare provider about the best vaccine option and timing for your vaccination. Currently, a COVID-19 vaccine is not expected to be available for young children until more studies are completed (see Q3). Those with SMA eligible to receive the vaccine, and all caregivers, should receive the approved COVID-19 vaccine to help protect those who may not be eligible to receive the vaccine at this time.

Are the COVID-19 vaccines safe?
The U.S. vaccine safety system ensures that all vaccines are as safe as possible. Based on the results from clinical trials of tens of thousands of adults, a determination is made about the efficacy and safety of the COVID-19 vaccine in the populations studied. After the FDA approves each vaccine, the federal vaccine advisory committee will review available data before making vaccine recommendations to U.S. Centers for Disease Control and Prevention (CDC).

How do I know which COVID-19 vaccine is the right one for me or my child to take?
All approved vaccines will be deemed effective and safe, and getting vaccinated to prevent COVID-19 is the number one priority. When available to people with SMA, we recommend speaking with your healthcare provider about the best vaccine option and timing for your vaccination. Currently, a COVID-19 vaccine is not expected to be available for young children until more studies are completed, so follow-up with your healthcare provider will be needed once one is available. In the interim, individuals with SMA and their families/caregivers should receive the COVID-19 vaccine when eligible.

What are the side effects of the COVID-19 vaccine?
The Pfizer vaccine has published the results of the COVID-19 clinical trial to date (Pollack FP et al. NEJM 2020). The most common side effects were short-term, including mild-to-moderate pain at the injection site, fatigue, and headache. Serious side effects incidence was low and was similar in both the placebo and treatment groups.

Will these vaccines interfere with receiving gene replacement therapy?
The current vaccines under FDA consideration, including the vaccine approved on December 11, 2020, do not interfere with any SMA treatment. Vaccines that are based on adenovirus also do not interact or interfere with the current SMA treatments. If a vaccine is based on Associated Adenovirus (AAV), there is a possibility for cross reactivity.

When I am eligible, where do I need to go to get my COVID-19 vaccination?
When a COVID-19 vaccine is available for people with SMA, we recommend speaking with and visiting your healthcare provider to be vaccinated. They know your medical history and can guide you in making decisions about which vaccine to get and timing.

What should I do to stay safe from COVID-19 while I am waiting for a vaccine to be made available?
While the current progress with vaccines for COVID-19 looks to be very positive, Cure SMA still strongly recommends that the SMA community continue the highest levels of isolation, given we are in the middle of the winter virus season and at the highest peak so far in this pandemic. At a minimum, you should cover your mouth and nose with a mask when around others, avoid close contact with people who are sick, stay 6 feet away from others, avoid crowds, and wash your hands often.

After I am vaccinated for COVID-19, will I be able to start being around people again?
Unfortunately, there is not enough information currently available to say if or when the CDC will stop recommending that people wear masks and avoid close contact with others to help prevent the spread of COVID-19. Experts need to understand more about the protection that COVID-19 vaccines provide before making that decision. If you are around people after you have been vaccinated, we still recommend diligent masking and social distancing.

Will I have to pay for the COVID-19 vaccine when I get it?
According to the CDC, vaccine doses purchased with U.S. taxpayer dollars will be given to the American people at no cost.

Kenneth Hobby,
President, Cure SMA

Mary Schroth, M.D. 
Chief Medical Officer, Cure SMA

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© Cure SMA | Email Preferences

Dear Friend,

Although the week is nearing its close, we''re happy to have the opportunity to contnue connecting with everyone virtually. Thank you to all who have joined the 2020 Virtual SMA Conference so far!

Use the links below to register for today''s free webinar events. Please note, if you have not already registered, you will need to register for each individual event that you are interested in attending.

View the 2020 Virtual SMA Conference booklet for the week''s details ▶

Thursday, June 11, 2020 

(All times in Central Time) 

10:00 a.m. - 10:45 a.m.
Oral Placement Therapy (OPT): Using Muscle-Based Therapy with Clients with the Diagnosis of SMA

11:30 a.m. - 12:15 p.m.
Breathing Basics

2:00 p.m. - 2:45 p.m.
SMA Clinical Trials

3:30 p.m. - 4:15 p.m.
Women''s Health in SMA

What Does Unity Mean to You and Your Loved Ones?  

This week, we''re asking you to share pictures of what unity looks like to you and those you love. To submit your photos or messages, please email us at stories@curesma.org or tag your post on social with #VirtualSMAConference!

Be sure to follow Cure SMA on Facebook, Twitter and Instagram - you may see yourself on our social media channels! 

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Dear Friend,

We want to thank everyone who joined us for this week''s first installment of the Summit of Strength Virtual Webinar series, "Supporting the Emotional Health of our SMA-Affected Families During Uncertain Times". The incredibly thoughtful webinar provided insights for everyone and featured Al Freedman, Ph.D., SMA parent and Child & Family Psychologist at Freedman Counseling Associates, as well as Jennifer Miller-Smith, SMA parent and representative from AveXis. 

If you were not able to join us, this is a recording worth revisiting and is now available to view online. 

Be sure to join us for next week''s second webinar in the Summit of Strength Virtual Webinar series, as we focus on Physical Therapy At Home. 

Thank you to the National Presenting Sponsors for the 2020 Summit of Strength Programs: AveXis, Biogen, and Genentech. We would also like to recognize Platinum Sponsor, Scholar Rock, for its support.

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

Cure SMA would like to remind you that registration is still open for the 2020 Virtual SMA Conference, which will be held from Monday, June 8th - Friday, June 12th. We greatly look forward to virtually uniting as a community starting tomorrow! It is our #SMAConferenceAtHome.

This year’s conference will continue the tradition of bringing together the SMA community to lend each other support and strength, and to learn more about the latest advances in treatments, research, advocacy, and care. The virtual gathering will include both webinar-based workshops and video networking opportunities.

The 2020 Virtual SMA Conference will kick off tomorrow morning with an opening session featuring Cure SMA President, Kenneth Hobby, along with board chairs Richard Rubenstein and Nick Farrell. We will offer a newly diagnosed program, and workshops on a wide range of topics including aquatic therapy, financial planning, education, medical management, and mental health. The conference will also offer four networking sessions for adults with SMA and SMA grandparents, as well as three industry symposiums for families.

To register for these free and live webinar and networking events, visit the Virtual SMA Conference webpage. Please note, you will need to register for each individual event that you are interested in attending. Registration confirmations will come with a specific link that can only be used for that event. Check out the full 2020 Virtual SMA Conference booklet, which offers further details on each event. 

Finally, Cure SMA is also encouraging the whole SMA community—individuals and families affected by SMA, researchers, and healthcare providers—to participate in a special end-of-conference celebration. With the goal of showing the strength of the SMA community, we would like everyone to share the many ways in which we are still united during this truly unconventional conference. Think about what represents “unity” for you and your loved ones, then share this symbol on your social channels (Facebook, Twitter, Instagram) Friday evening, June 12th at 7:00 p.m. CT, using the #SMAConferenceAtHome and #CureSMA. We look forward with seeing the SMA community come together as we show how we are “United, At Home.”

If you have any questions, feel free to contact conference@curesma.org.

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

We want to thank everyone who joined us for today''s Coronavirus (COVID-19) Community Webinar. We hope that the webinar helped answer questions and addressed concerns regarding the Coronavirus (COVID-19) public health crisis. During this time, Cure SMA would like to continue to be a resource for the entire SMA community.

If you were not able to join us, a recording of the webinar is now available to view online. 

Please note: All discussions around support and care items are intended for U.S. audiences only.

For more support and questions related to COVID-19, visit www.curesma.org/covid19. 

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

As news about the Coronavirus (COVID-19) continues to evolve, Cure SMA has now decided to cancel all events (i.e., galas, dinners, Walk-n-Rolls, Summits of Strength) through the end of May. This decision took into account the most recent recommendation from the Centers for Disease Control and Prevention (CDC) to limit or cancel all gatherings of 10 people or more for the next 8 weeks. Recognizing the time and effort that our event organizers and families put into planning and preparing for these events in advance, at this time we hope to make sure that the focus of the SMA community remains on staying healthy.

We continue to evaluate the potential impact of COVID-19 on the upcoming 2020 Annual SMA Conference in Orlando, Fla. (June 11-14, 2020). The decision will be made in the best interest of the entire SMA community, as your health, safety, and well-being continue to be our top priority.

In the meantime, if you have already registered for the 2020 Annual SMA Conference and booked your travel and accommodations, we recommend that you keep your plans in place. In the event of a cancellation of the 2020 Annual SMA Conference:

• All Cure SMA registration fees will remain fully refundable.
• Hotel reservations within the Cure SMA hotel room blocks can be cancelled up to six (6) days prior to the arrival date to receive a refund of the deposit.
• Many airlines are now offering no change fees on booking new travel (please look at each airline policy closely to see what applies for you).
• Cure SMA will work to support and cover non-refundable expenses that registrants may incur as a result of a Conference cancellation.

For information on the COVID-19 and general prevention measures, please refer back to Cure SMA’s Community Statement from March 9.

If you have any other questions, please reach out to info@curesma.org.

Kenneth Hobby
President, Cure SMA

Mary Schroth, M.D.
Chief Medical Officer, Cure SMA

Jill Jarecki, Ph.D.
Chief Scientific Officer, Cure SMA

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Dear Friend,

On Friday, July 31, at 1:00 p.m. ET/12:00 p.m. CT/11:00 a.m. MT/10:00 a.m. PT, Cure SMA will hold its next COVID-19 SMA Community Webinar (watch the March 27 webinar here and the May 28 webinar here). We would like to continue to be a resource for the entire SMA community as we manage the daily challenges of the pandemic, and that means hearing your latest questions.

REGISTER ➱

You will hear from the Cure SMA team including Kenneth Hobby, President of Cure SMA; Mary Schroth, M.D., Chief Medical Officer; and Colleen McCarthy O''Toole, VP of Events & Family Support.

The agenda for the webinar will be shaped by your questions submitted in advance (e.g., back to school/work, IEPs, community experience with COVID, etc.). Please submit your questions when you register or by emailing webinar@curesma.org by end of day Friday, July 24. We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

Last week, Cure SMA hosted its first-ever Virtual SMA Conference. Even in these times of physical distancing, affected individuals, families, healthcare professionals, and researchers came together to show the strength and unity of the SMA community by attending virtual sessions, networking opportunities, and celebrations. By hosting the conference virtually, Cure SMA confirmed that no challenge will slow us down and we remain focused on continuing our mission.

Without the dedication of our supporters, partners, and committed donors like you, we would not be able to provide resources like the Annual SMA Conference that the SMA community depends on. We ask you to please consider making a gift to Cure SMA today.

To show our thanks, every donor that makes a gift of $50 or more by June 28 will have their name added to our Donor Appreciation Wall. Stay tuned in for more information as our heart continues to grow with love and appreciation for our loyal donors.

Thank you in advance for your support. 

Very Best,

Erin Kelly 
Vice President of Development, Cure SMA 

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© Cure SMA | Email Preferences

Dear Friend,

While this year has been different in many ways, one thing has not changed: Cure SMA continues to make unprecedented progress and advance the life-changing research, care, and support programs that are shaping a stronger future for all individuals with spinal muscular atrophy (SMA) and their families.

Whether we are growing our local presence to ensure access to approved treatments, creating customized programs that help meet the daily needs of those with SMA, advancing the development of combination therapies that increase strength and enable independence, or leading the way to an evidence-based standard of care for SMA, Cure SMA continues to advance our mission. As we head into 2021, we need your help to expand these programs that support adults, teens, and children with SMA.

Continuing today, and running through the end of the day on, December 13, anyone who donates $100 or more will be entered for a chance to win a wonderful Cure SMA Holiday Swag Bag filled with items including a Cure SMA License Plate Frame, Hand Blown Glass Ornament, Embroidered Plush Blanket, Stainless Steel Travel Mug, and a Cure SMA Striped Pom Beanie. Make a gift today and tell your friends and family about this exciting opportunity!

Thank you in advance for your support. 

Very Best,

Erin Kelly
Vice President, Development 

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

A mid-week hooray and thank you to all who joined us for Day Two of the Virtual SMA Conference! We hope you found yesterday''s webinars and sessions enjoyable. 

Use the links below to register for today''s free webinar events. Please note, if you have not already registered, you will need to register for each individual event that you are interested in attending.

View the 2020 Virtual SMA Conference booklet for the week''s details ▶

Wednesday, June 10, 2020 

(All times in Central Time) 

10:00 a.m. - 10:45 a.m.
Medical Management of Adults with SMA

11:30 a.m. - 12:15 p.m.
How to Find, Hire and Manage Your PCAs

1:00 p.m. - 1:45 p.m.
Relevant Assistive Technology Tools for Individuals with SMA: Where We''ve Come and Where We’re Going!

3:00 p.m. - 4:00 p.m.
Family Symposium, Sponsored by AveXis: Zolgensma (onasemnogene abeparvovec-xixo) Clinical Study Update

7:00 p.m. - 9:00 p.m.
Adults with SMA Social at Home, sponsored by Biogen 
*To register for this networking event on Zoom, please email familysupport@curesma.org.

Share Your Photos With Cure SMA on Social Media 

We want to see how you''re tuning into the #VirtualSMAConference!

Be sure to follow Cure SMA on Facebook, Twitter and Instagram - you may see yourself on our social media channels! 

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

Cure SMA has released the agenda and registration details for the 2020 Virtual SMA Conference, which will be held from Monday, June 8th - Friday, June 12th. We greatly look forward to virtually uniting as a community in just a few short weeks! It is our #SMAConferenceAtHome.

This year’s conference will continue the tradition of bringing together the SMA community to lend each other support and strength, and to learn more about the latest advances in treatments, research, advocacy, and care. The virtual gathering will include both webinar-based workshops and video networking opportunities.

The 2020 Virtual SMA Conference will kick off with an opening session featuring Cure SMA President, Kenneth Hobby, along with board chairs Richard Rubenstein and Nick Farrell. We will offer a newly diagnosed program, and workshops on a wide range of topics including aquatic therapy, financial planning, education, medical management, and mental health. The conference will also offer four networking sessions for adults with SMA and SMA grandparents, as well as three industry symposiums.

To register for these free webinar and networking events, visit the Virtual SMA Conference webpage. Please note, you will need to register for each individual event that you are interested in attending. Registration confirmations will come with a specific link that can only be used for that event.

Keep an eye out for the full 2020 Virtual SMA Conference booklet, which will provide further details on each event.

Finally, Cure SMA is also encouraging the whole SMA community—individuals and families affected by SMA, researchers, and healthcare providers—to participate in a special end-of-conference celebration. With the goal of showing the strength of the SMA community, we would like everyone to share the many ways in which we are still united during this truly unconventional conference. Think about what represents “unity” for you and your loved ones, then share this symbol on your social channels (Facebook, Twitter, Instagram) Friday evening, June 12th at 7:00 p.m. CT, using the #SMAConferenceAtHome and #CureSMA. We look forward with seeing the SMA community come together as we show how we are “United, At Home.”

If you have any questions, feel free to contact conference@curesma.org.

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

The day is finally here! Today we kick off Cure SMA''s first-ever Virtual SMA Conference!

Use the links below to register for today''s free webinar events. Please note, if you have not already registered, you will need to register for each individual event that you are interested in attending.

View the 2020 Virtual SMA Conference booklet for the week''s details ▶

Monday, June 8, 2020 

(All times in Central Time) 

10:00 a.m. - 11:00 a.m.
Welcome: Opening Session for the 2020 Virtual SMA Conference

11:30 a.m. - 12:15 p.m.
2020 SMA Clinical Care and Research Updates

1:00 p.m. - 2:00 p.m.
Virtual Session for Newly Diagnosed Families

3:00 p.m. - 4:00 p.m.
Family Symposium, Sponsored by Genentech

6:00 p.m. - 7:00 p.m.
*Grandparent Virtual Mingle

7:00 p.m. - 9:00 p.m.
*Adults with SMA Social At Home, sponsored by Biogen 

*To register for these events, please email familysupport@curesma.org. 

Follow Cure SMA on social media!

Be sure to follow Cure SMA on Facebook, Twitter and Instagram for the latest conference updates.

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

Cure SMA is excited to reunite the SMA community for the 2021 Annual SMA Conference in Austin, Texas from Thursday, June 10 to Sunday, June 13, 2021.

If the unexpected circumstances of 2020 have shown us anything, it is how impactful this one week of the year is for families, individuals, researchers, and medical professionals in the SMA community. We cannot wait to welcome you back as we gather next June in Texas. The JW Marriott Austin will host the 2021 Annual SMA Conference and the Annual International SMA Research & Clinical Care Meeting. The hotel is centrally located in Austin’s lively downtown neighborhood with a variety of restaurants, live music, and entertainment venues.

Each year, Cure SMA sponsors a conference to bring together the leading SMA researchers, clinicians, individuals, and families affected by SMA. As always, the SMA Research & Clinical Care Meeting runs alongside the Annual SMA Conference. We have been hosting the Annual SMA Conference since 1988, and it has become a weekend filled with a wide variety of activities, including the following:

• Educational workshops.
• Keynote sessions with leading researchers and clinicians.
• Family-friendly poster session.
• Memorable children’s program.
• Meet and greet with a family fun fest.
• Teen and adult social activities.
• PJ party and movie night.
• Dance party.

This is the largest conference in the world for those affected by SMA, and for those involved in providing support and care for SMA patients. It brings together researchers, healthcare professionals, affected individuals, and families to network, learn, and collaborate. All these activities provide opportunities for individuals and families to connect and interact, while also receiving first-hand updates from researchers and clinicians.

More details about the 2021 Annual SMA Conference and SMA Research & Clinical Care Meeting will be announced in the upcoming months and registration will launch in the fall of 2020. We look forward to seeing you in Austin!

If you have any questions, please contact conference@curesma.org.

800-886-1762 | info@curesma.org | curesma.org

© Cure SMA | Email Preferences

Dear Friend,

SMA Awareness Month is winding down but we have one last opportunity for you to get involved. Throughout August, Cure SMA has engaged with the SMA community, shined a spotlight on our efforts to support those living with SMA, and leveraged key partnerships to build on the progress we have made. With your help today, we can finish the month strong and generate the excitement and passion needed to carry this momentum throughout the year.

Cure SMA’s Chief Medical Officer, Dr. Mary Schroth, is asking you to help lead this final push, and she is willing to go to great lengths to get you involved…

In honor of SMA Awareness Month, Dr. Schroth has agreed to dye her hair purple if at least 50 people make donations to Cure SMA this weekend. The gifts that we receive will help expand access to available treatments and improve care for those who need it most. Can we count on you to help ensure that Dr. Schroth dyes her hair purple? If so, please donate today.

Thank you in advance for your support. 

Very Best,

Erin Kelly
Vice President, Development 

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Dear Friend,

We hope you had a happy and healthy Thanksgiving weekend. With #GivingTuesday, just a few days away, we wanted to make sure you have an opportunity to participate in any of our planned charitable activities that benefit Cure SMA.

Continuing for the next three days, Cookies4Cures—a nonprofit founded by 10-year-old Dana Perella—is matching every donation to Cure SMA through November 30, up to $10,000! By making a gift to our Year End Campaign, you can double your impact for the spinal muscular atrophy (SMA) community. This means you can advance twice the number of breakthroughs in research, track twice the number of changes in treatment progression, and provide twice the number of support packages to individuals with SMA and their families.

Another match opportunity to consider is on #GivingTuesday, where The Johnny III Foundation, created in memory of John Truett Whitley III, is matching all donations up to $2,500. And, as if you needed another reason to give, if you make a gift of $100 or more on December 1, you will be entered to win a limited-edition Cure SMA Ugly Holiday Sweater. This give-away is one-of-kind, and you don’t want to miss out on your chance to win an original Cure SMA Ugly Holiday Sweater.

Whether you choose to make a gift today, or participate on #GivingTuesday, you will help advance the research, care, and support programs that are shaping a stronger future for everyone affected by SMA.

Thank you in advance for your support. 

Very Best,

Erin Kelly
Vice President, Development 

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Dear Friend,

ICYMI: On Friday, July 31, at 1:00 p.m. ET/12:00 p.m. CT/11:00 a.m. MT/10:00 a.m. PT, Cure SMA will hold its next COVID-19 SMA Community Webinar (watch the March 27 webinar here and the May 28 webinar here). We would like to continue to be a resource for the entire SMA community as we manage the daily challenges of the pandemic, and that means hearing your latest questions.

REGISTER ➱

You will hear from the Cure SMA team including Kenneth Hobby, President of Cure SMA; Mary Schroth, M.D., Chief Medical Officer; and Colleen McCarthy O''Toole, VP of Events & Family Support.

The agenda for the webinar will be shaped by your questions submitted in advance (e.g., back to school/work, IEPs, community experience with COVID, etc.). Please submit your questions when you register or by emailing webinar@curesma.org by end of day TODAY, Friday, July 24. We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

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Dear Friend,

Cure SMA has been hosting the Annual SMA Conference since 1988, bringing together leading researchers, clinicians, affected individuals, and families living with SMA. The weekend has traditionally been filled with a wide variety of opportunities to learn about the latest in treatments, research, advocacy, care, and support. But most of all, it was a time to connect and interact with the whole SMA community through engaging events like the Meet & Greet, PJ party, adult networking, and more.

Although we are unable to come together in-person this summer, Cure SMA is thrilled to announce it will offer a special Conference-like program to allow individuals and families affected by SMA the chance to be “United, At Home.” The 2020 Virtual SMA Conference will take place during the planned Annual SMA Conference week—June 8-12, 2020.

Cure SMA will host nearly two dozen presentations, networking opportunities, and sponsor symposia throughout the week, kicking off with a traditional opening session on Monday, June 8. And since the SMA Research & Clinical Care Meeting will also be happening in parallel to the Virtual SMA Conference, we are planning a special end-of-week virtual celebration on Friday, June 12, which will bring together researchers, clinicians, and affected individuals and families to demonstrate the unity of the full SMA community—despite the uncommon times we are living in.

More information, including the full agenda and how to register for virtual sessions, will be announced soon. We look forward to meeting together virtually!

If you have any questions about the #SMAConferenceAtHome, please contact conference@curesma.org.

Thank you to the National Presenting Sponsors for the 2020 Annual SMA Conference: AveXis, Biogen, and Genentech. We would also like to recognize the support of our Visionary Sponsor, Ionis; Platinum Sponsor, Scholar Rock; and Silver Sponsor, Accredo.

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Dear Friend,

This is a final reminder to register for tomorrow''s Cure SMA COVID-19 Spinal Muscular Atrophy (SMA) Community Webinar at 1:00 p.m. ET/12:00 p.m. CT/11:00 a.m. MT/10:00 a.m. PT. 

REGISTER ➱

The agenda for the webinar is focused on the upcoming flu season, vaccine updates, and Cure SMA''s latest advocacy efforts and new support programs for individuals with SMA and their families. We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

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Dear Friend,

Thank you again to everyone who joined us for the 2020 Virtual SMA Conference! It was an impactful week of online workshops, networking, and community. If you weren''t able to tune in for the live events, we''ve got you! Recordings of 16 online sessions are now available to view on our YouTube channel.

More than 6,600 participants from 77 different countries who attended the 28 offered live events. Check out the full 2020 Virtual SMA Conference recap here.

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Dear Friend,

On Thursday, October 29, at 1:00 p.m. ET/12:00 p.m. CT/11:00 a.m. MT/10:00 a.m. PT, Cure SMA will hold its next COVID-19 SMA Community Webinar. We would like to continue to be a resource for the entire SMA community as we manage the daily challenges of the pandemic, and that means hearing your latest questions.

REGISTER ➱

You will hear from the Cure SMA team including Kenneth Hobby, President; Mary Schroth, M.D., Chief Medical Officer; Colleen McCarthy O''Toole, VP of Family Support; and Maynard Friesz, VP of Advocacy. 

The agenda for the webinar will be shaped by your questions submitted in advance (e.g., continued isolation, upcoming flu season, vaccine updates, community experiece with COVID, etc.). Please submit your questions when you register or by emailing webinar@curesma.org by end of day Wednesday, October 21. We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

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Dear Friend,

This is a reminder to register for tomorrow''s webinar at 11:30am CST (9:30am PST/10:30am MST/12:30pm EST). This SMA community webinar will help answer COVID-19 questions and address concerns we have heard in recent days and weeks. During this time, Cure SMA would like to continue to be a resource for the entire SMA community.

Register ▶

The webinar will provide the community with up-to-date virus information and Cure SMA activities and programs created in response to the COVID-19 public health crisis. Hear from Cure SMA leadership team, including Kenneth Hobby, President of Cure SMA; Mary Schroth, M.D., Chief Medical Officer; and Colleen McCarthy O''Toole, VP of Events and Family.

The 60-minute webinar will also include a Q&A session based on questions submitted in advance of the call. We encourage you to submit your questions when you register or by emailing webinar@curesma.org, so that we can plan to answer as many as possible. We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

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Dear Friend,

Cure SMA has been proactive in our response to the COVID-19 pandemic. Our COVID-19 Information Center offers the most up-to-date information and resources for the health needs of the entire SMA community. We have also migrated our in-person support programs to virtual platforms so these resources remain available to all affected individuals and families.

Cure SMA is uniquely prepared for this moment and while we provide these essential new services, we continue to focus on our mission. We are still investing in comprehensive research in treatment and care to create new breakthroughs for everyone with SMA, and providing the family support programs that our community depends on.

Today is #GivingTuesdayNow, a worldwide day of charitable giving created to help bring the global community together in response to COVID-19. If you are in a position to give, please consider donating to Cure SMA. Every dollar counts, and all donations will be used to help us support the SMA community.

Thank you in advance for your support. 

Very Best,

Erin Kelly 
Vice President of Development, Cure SMA 

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Dear Friend,

If you haven''t already done so, there is still time to register for Cure SMA''s next COVID-19 SMA Community Webinar on Friday, July 31, at 1:00 p.m. ET/12:00 p.m. CT/11:00 a.m. MT/10:00 a.m. PT. You will hear from the Cure SMA team including Kenneth Hobby, President of Cure SMA; Mary Schroth, M.D., Chief Medical Officer; and Colleen McCarthy O''Toole, VP of Events & Family Support.

REGISTER ➱

We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

P.S. - Check out the March 27 webinar here and the May 28 webinar here!

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Dear Friend,

It has been more than two months now since many states initiated shelter-at-home orders as a result of the worldwide COVID-19 pandemic. As the SMA community continues to manage these ever-evolving changes, Cure SMA remains committed to supporting all affected individuals and families.

On Thursday, May 28th, at 1:00 p.m. ET/12:00 p.m. CT/11:00 a.m. MT/10:00 a.m. PT, Cure SMA will host its second COVID-19 SMA Community Webinar (watch the March 27th webinar here). We would like to continue to be a resource for the entire SMA community as we manage the daily challenges of the pandemic, and that means hearing your latest questions.

REGISTER ➱

You will hear from the Cure SMA team including Kenneth Hobby, President of Cure SMA; Mary Schroth, M.D., Chief Medical Officer; Colleen McCarthy O''Toole, VP of Events & Family Support; and Maynard Friesz, VP of Policy & Advocacy.

The agenda for the 60-minute webinar will be shaped by your questions submitted in advance. Please submit your questions when you register or by emailing webinar@curesma.org by end of day Thursday, May 21st. We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

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Dear Friend,

Thank you to everyone who joined us this week and to those who have joined us in years past!

Over the last five days, we''ve had the opportunity to virtually connect with the entire SMA community — from individuals and families — to researchers and healthcare providers. Though we weren''t physically together to celebrate in Orlando, the bonds created from previous conferences and the ability to stay connected this week proved that we are a united community.

If you tuned in to any virtual sessions this week, we want your feedback! Please complete this quick survey. 

Save the Date: 2021 Annual SMA Conference in Austin, TX 

We are excited to reunite the SMA community for the 2021 Annual SMA Conference in Austin, Texas from Thursday, June 10 - Sunday, June 13, 2021. 

The JW Marriot Austin, centrally located in Austin’s lively downtown neighborhood, will host next year''s conference. More details, including registration, will be available in Fall 2020. 

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Please disregard if you are receiving this email for a second time; no new information is provided below. A glitch occurred in our system and we wanted to ensure our full community received this important information.

Dear Friend,

As coronavirus, also known as COVID-19, continues to spread in communities, Cure SMA has also been closely tracking the situation. The health, safety, and well-being of the entire SMA community is important to us. While community engagement is a longtime Cure SMA priority and several opportunities for involvement are fast approaching, we are cognizant of the need to protect those with SMA who may be more vulnerable. Because of this, Cure SMA has made the decision to cancel all events (i.e., galas, dinners, Walk-n-Rolls, Summits of Strength) for the remainder of March and is currently assessing the possibility of cancellation of events in April and beyond.

We are also carefully evaluating the potential impact on the upcoming 2020 Annual SMA Conference in Orlando, Fla. (June 11-14, 2020). With approximately 3 months until the start of the Conference, we are actively monitoring the impact of COVID-19 in the U.S. so we can make a decision that is in the best interest of the SMA community. Currently, we remain optimistic about hosting another successful and well-attended Annual SMA Conference this Summer.

Questions on Event Cancellations and Annual Conference Planning: 

1. What Cure SMA events will you be cancelling?
   Cure SMA has made the decision to cancel all events (i.e., galas, dinners, Walk-n-Rolls, Summits of Strength) for the remainder of March and is assessing whether to cancel events in April and beyond. If there are any changes to an event that you have already registered for, an email will be sent by Cure SMA with further details.
2. I am scheduled to attend a Cure SMA event that is cancelled. Will you be rescheduling it?
   At this time, we are not certain which, if any, events will be rescheduled. Cure SMA will alert event registrants as soon as we know more. In the meantime, if you have any questions related to a Cure SMA event, please reach out to info@curesma.org, noting your event name in the subject line.
3. Should I still plan to book my travel and accommodations for the Annual SMA Conference in Orlando this June? If I already made my plans, should I cancel my reservations?
   The Annual SMA Conference is not cancelled. Registration remains strong and we remain optimistic about hosting another successful and well-attended Annual SMA Conference this Summer. If you have already registered for the 2020 Annual SMA Conference and booked your travel and accommodations, we recommend that you keep your plans in place. For those who have not registered yet, we recommend that you register and continue booking your travel needs to ensure your space is reserved. Many airlines are offering deals for newly booked flights, where changes or cancellations can be made within a specific timeline.
4. If the Annual SMA Conference does get cancelled later, will I get a refund on my registration, travel, and accommodations?
   In the event of a cancellation of the 2020 Annual SMA Conference:

• All Cure SMA registration fees will remain fully refundable.
• Hotel reservations within the Cure SMA hotel room blocks can be cancelled up to six (6) days prior to the arrival date to receive a refund of the deposit.
• Many airlines are now offering no change fees on booking new travel (please look at each airline policy closely to see what applies for you).
• Cure SMA will work to support and cover non-refundable expenses that registrants may incur as a result of a Conference cancellation.

If you have any questions related to the Annual SMA Conference, please reach out to info@curesma.org. 

Information and Recommendations on COVID-19 

The SMA community’s concern has been rising as cases of COVID-19 increase in the U.S., primarily in urban areas. While discussions continue around future Cure SMA events, we would like to share some information on the disease and prevention measures for your easy reference.

What We Know: 

According to the Centers for Disease Control and Prevention (CDC), the immediate risk of exposure to the COVID-19 is thought to be low. However, in places where ongoing community spread has been reported, people are at elevated risk of exposure.

• The virus is spread by respiratory droplets produced when an infected person coughs or sneezes. These droplets can land in the eyes, noses, or mouths of people who are nearby, or even be inhaled into the lungs.
• People are thought to be most contagious when they are most symptomatic (i.e., the sickest) but can be contagious early in course of illness.
• The elderly and adults with underlying health conditions are at increased risk for severe disease, while, to date, children seem to present with milder disease.
• There is no approved treatment or vaccine for this virus.
• Masks should be worn by those with respiratory symptoms and may be helpful to control spread of infection. Wearing masks by healthy people is not recommended. Masks are necessary for the protection of healthcare providers.
• Typical symptoms may appear 2 to 14 days after exposure, presenting as fever, cough, and shortness of breath.

Protecting Yourself and Your Family: 

The best way to protect against respiratory illness, including COVID-19, is to continue to practice good health habits. 

• Stay calm. Concerns about a novel virus infection outbreak can lead to fear and anxiety. Health professionals, and we at Cure SMA, are here to help alleviate these fears.
• Avoid others who are sick.
• Frequently wash hands with soap and water for at least 20 seconds or use alcohol-based hand sanitizer with at least 60% alcohol base.
• Avoid touching eyes, nose, and mouth.
• Cover coughs and sneezes.
• Disinfect surfaces regularly.
• If traveling by air, wipe down all surfaces in the plane with antibacterial wipes designed for surfaces. The key is allowing the surface to airdry following wiping the surface with an antibacterial wipe.
• Avoid shaking hands, hugging, and kissing.
• Avoid large gatherings.
• Avoid travel to high risk locations.
• If you become ill, stay home, call your doctor’s office to discuss symptoms, and try to stay out of healthcare facilities.

Questions on COVID-19:

1. Is the SMA community at a higher risk for contracting COVID-19?
   The elderly and adults with underlying health conditions are at the greatest risk for severe disease. While children seem to have milder disease, it is not known whether children with underlying health conditions, such as SMA, are at increased risk for severe disease. However, in general, anyone with SMA is at increased risk for more severe disease due to the general increased fatigue associated with viral illness.
   
   If you have questions about SMA treatment decisions or needs, contact your healthcare provider and/or care team. They can advise on the safety of local healthcare facilities and if changes to your treatment timelines are warranted. For scheduled appointments and treatments, you may consider asking to be placed in a room upon arrival to avoid sitting in the waiting room.
2. Should I take my child out of school?
   Please discuss your concerns about exposure to COVID-19 with your school administrators. Alternatives to attending school in person may be available, including virtual or e-learning classrooms. Your school can advise about the education options available under these circumstances.

This is rapidly evolving situation and we recommend that the SMA community follow the guidance of the CDC. If you have any specific personal medical questions related to the COVID-19, please contact your healthcare provider.

Kenneth Hobby
President, Cure SMA

Mary Schroth, M.D.
Chief Medical Officer, Cure SMA

Jill Jarecki, Ph.D.
Chief Scientific Officer, Cure SMA

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Dear Friend,

Cure SMA has always been about creating community. During these exceptional times, we know individuals and families with SMA need us now more than ever. That is why Cure SMA is excited to announce the launch of our Virtual Walk-n-Roll and Virtual Team Cure SMA programs!

Although we cannot gather physically, we can create connections by networking, sharing stories, and having fun together from home. Whether you like to run, walk, roll, or just show your support for Cure SMA, we have something in store for you! Join one of our new programs today.

By supporting Cure SMA, you will help fund programs and research that continue to change the course of SMA for everyone affected. We hope you will join us as we unite to raise awareness for SMA. Find out more about virtual events by contacting us at fundraising@curesma.org.

The Virtual Walk-n-Roll is a new way to engage with Cure SMA from wherever you are even if a Walk-n-Roll was not planned in your area. Whether you plan to walk or roll around your neighborhood or your kitchen island, you will be part of a movement to make an impact with Cure SMA supporters across the country. To celebrate the efforts of the entire community, we will host the first-ever Cure SMA Virtual Walk-n-Roll Celebration Ceremony on May 30, 2020, at 11 a.m. EDT/10 a.m. CDT/9 a.m. MDT/8 a.m. PDT on Cure SMA’s YouTube channel. Save the date and be on the lookout for more details!

There is no registration fee to participate in the Cure SMA Virtual Walk-n-Roll and all are welcome! However, if you choose to make a self-donation or participate in fundraising activities, you qualify to earn fun Cure SMA incentives, including a limited-edition Virtual Walk-n-Roll t-shirt.

Join our Cure SMA Virtual Walk-n-Roll group on Facebook and learn more on our Virtual Kick-off stream on Tuesday, April 28 at 1 p.m. EDT/12 p.m. CDT/11 a.m. MDT/10 a.m. PDT.

Virtual Team Cure SMA is a new endurance program that offers runners and cyclists—both currently supporting Team Cure SMA or just thinking about it—a way to participate in races virtually while sharing their results and accomplishments. We want you to achieve your personal fitness goals, whatever they may be, and are here to support your fitness journey by providing race training plans that anyone can take advantage of from the convenience of home.

There is no fundraising requirement to participate, but athletes can earn awesome Team Cure SMA endurance gear if they choose to fundraise and reach milestones. Team Cure SMA is a group dedicated to taking on fun fitness challenges and we are here for you every step of the way, even virtually!

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Dear Friend,

Thank you to all who joined us for Day One of the Virtual SMA Conference! We hope you found yesterday''s webinars and sessions enjoyable. 

Use the links below to register for today''s free webinar events. Please note, if you have not already registered, you will need to register for each individual event that you are interested in attending.

View the 2020 Virtual SMA Conference booklet for the week''s details ▶

Tuesday, June 9, 2020 

(All times in Central Time) 

10:00 a.m. - 10:45 a.m.
Aquatic Physical Therapy

11:30 a.m. - 12:15 p.m.
Life After Diagnosis: 25 Years of Lessons Learned

2:00 p.m. - 3:00 p.m.
Family Symposium, Sponsored by Biogen: Treatment and Multidisciplinary Care in SMA

7:00 p.m. - 9:00 p.m.
*Adults with SMA Lounge Night, sponsored by Biogen 

*To register for these events, please email familysupport@curesma.org. 

Share Your Photos With Cure SMA on Social Media 

We want to see how you''re tuning into the #VirtualSMAConference!

Be sure to follow Cure SMA on Facebook, Twitter and Instagram - you may see yourself on our channels! 

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Dear Friend,

For the past two years, Cure SMA’s Summit of Strength program has brought together thousands of SMA community members for the opportunity to network and learn about the latest advances in treatment, care, advocacy, and support. Considering event cancellations across the country due to the ongoing pandemic, Cure SMA is excited to announce the kickoff of our Summit of Strength Virtual Webinar Series.

The virtual webinars will start in May and run through the summer, bringing to your home on most Wednesdays experts who will speak on a variety of educational topics tailored to help individuals and families with SMA during this life in quarantine. These presentations will include family support and patient care topics like those that may have been shared at our in-person summits. Also, during each webinar, attendees will hear a brief presentation from one our Summit of Strength presenting sponsors: AveXis, Biogen, or Genentech.

The first installment of the Summit of Strength Virtual Webinar Series will be next Wednesday, May 13th, focused on Supporting the Emotional Health of Our SMA-Affected Families During Uncertain Times. Al Freedman, Ph.D., an SMA Parent and Child & Family Psychologist at Freedman Counseling Associates, will present, as will Jennifer Miller-Smith from AveXis, speaking on “Adapting SMA Life During the Time of COVID-19.” Please click here to register for this webinar and the chance to submit a question in advance.

Despite the new reality of postponed events, remote working, and social distancing, Cure SMA’s work for the SMA community continues as we remain committed to providing families the support they need for today. While we look forward to returning to in-person Summits of Strength when we are able, you can still engage with Cure SMA Chapter Leaders and connect with your local SMA community.

If you have any questions about the Virtual Summit of Strength Webinars, please contact familysupport@curesma.org.

Thank you to the National Presenting Sponsors for the 2020 Summit of Strength Program: AveXis, Biogen, and Genentech. We would also like to recognize Platinum Sponsor, Scholar Rock, for its support.

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Dear Friend,

Cure SMA is pleased to announce that the 2020 Virtual SMA Conference booklet is now available for download. The booklet includes important details on the Virtual SMA Conference, including:

1. A full agenda with event descriptions and speaker bios for the 16 virtual topical workshops. Don’t forget to register for each individual event that you are interested in attending!

2. Details about special evening networking events, which you can register for by contacting familysupport@curesma.org.

• Monday, June 8th from 6:00pm to 7:00pm CT, we will host a Grandparent Virtual Mingle, where SMA grandparents can connect and chat on Zoom.
• Monday, June 8th from 7:00pm to 9:00pm CT, a Biogen-sponsored Adults with SMA Social on Zoom will provide a friendly platform to socialize from home.
• Tuesday, June 9th from 7:00pm to 9:00pm CT, a Biogen-sponsored Adults with SMA Lounge Night on Zoom will allow adults with SMA the chance to virtually hangout.
• Wednesday, June 10th from 7:00pm to 9:00pm CT, a Biogen-sponsored Adults with SMA Social on Zoom will provide a friendly platform to socialize from home.

3. Registration details for the Industry-sponsored family-focused symposia.

• Monday, June 8th from 3:00pm to 4:00 pm Family Symposium, Sponsored by Genentech. SMA My Way: When Resilience is a Superpower - Support Systems, Changing Perspectives and Focusing on the Positives with Shane Burcaw and Hannah Aylward
• Tuesday, June 9th from 2:00pm to 3:00pm Family Symposium, Sponsored by Biogen. Treatment and Multidisciplinary Care in SMA
• Wednesday, June 10th from 3:00pm to 4:00pm Family Symposium, Sponsored by AveXis. ZOLGENSMA® (onasemnogene abeparvovec-xioi) Clinical Study Update

4. Information about our wonderful sponsors of the 2020 Virtual SMA Conference. 

Be sure to follow Cure SMA on Facebook, Twitter, and Instagram for the latest conference updates. To connect with other attendees and families, join the Annual SMA Conference Facebook Group.

We’re expecting an enthusiastic showing of families, individuals, healthcare providers, professionals, and SMA researchers who will be joining our week of education and community-building. We hope to see you online as we show how we are “United, At Home.”

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Dear Friend,

To all our SMA families, volunteers, sponsors, and supporters, we want you to know that Cure SMA is here for you.

Cure SMA remains vigilant in monitoring and responding to the needs of the SMA community, as your health, safety, and well-being is our top priority.

The following FAQs are designed to help address key concerns expressed by members of the SMA community about COVID-19 and provide additional information about how you can protect yourself and your family. We encourage you to refer to the Centers for Disease Control and Prevention (CDC) for the most up-to-date information.

To read all Cure SMA communications and guidance to date, visit the Cure SMA Coronavirus (COVID-19) Information Center.

If you have any questions, please email info@curesma.org. We will also be announcing a Community Webinar in the coming weeks, so stay tuned for more information via email and social media.

Frequently Asked Questions

Are people with SMA at increased risk for COVID-19 infection? 

Evidence has shown that people with underlying health conditions may be at greater risk of serious illness from COVID-19. The CDC has issued guidelines for people who are at higher risk. General recommendations include:

• Stay at home as much as possible to further reduce your risk of being exposed. As possible, other members in the household should also stay at home.
• Stock up on supplies, such as antibacterial wipes, medications, hand sanitizer, equipment supplies, formula, and feeding supplies.
• Take precautions to keep space between yourself and others.
• When you go out in public, keep away from others who are sick, limit close contact, and wash your hands often.
• Avoid crowds crowds or large gatherings.
• Avoid cruise, non-essential air travel, and travel to high risk locations. 

Cotact your healthcare provider for specific questions about your health. Check the CDC website for complete guidelines and up-to-date information. 

How can I protect myself and my family? 
The best way to protect against respiratory illness, including COVID-19, is to continue to practice good health habits.

• Avoid others who are sick.
• Frequently wash hands with soap and water for at least 20 seconds or use alcohol-based hand sanitizer with at least 60% alcohol base.
• Avoid touching eyes, nose, and mouth.
• Cover coughs and sneezes with tissue, throw away the tissue and wash hands.
• Disinfect surfaces regularly.
• Avoid shaking hands, hugging, and kissing.
• If you become ill, stay home and call your doctor’s office to discuss symptoms. Try to stay out of healthcare facilities.

Should I wear a facemask?
Masks should be worn by those with respiratory symptoms and may be helpful to control spread of infection. If you are not sick, you do not need to wear a facemask unless you are caring for someone who is sick, and they are not able to wear a facemask. Facemasks may be in short supply and they should be saved for caregivers and healthcare professionals.

Is it safe for me to go to work or school?
We recognize that every individual''s circumstances are unique. Questions about your personal risk factors should be directed to your healthcare team, who can help you determine if you should take additional precautions.

• School: We strongly recommend that children with SMA stay home from school. Virtual learning or other remote options should be considered in all cases, and most schools across the country are accommodating this need.
• Work: We strongly recommend that adults with SMA work remotely whenever possible.

Is it safe for me to continue going to clinic visits?
People with SMA should continue their usual care routine, unless otherwise guided by your healthcare provider. Routine well-visits should be conducted by telehealth approaches or postponed whenever possible. Healthcare teams have been asked to think creatively—such as phone check-ins, avoiding time in waiting rooms—to avoid unnecessary risk of exposure and pressure on their staff and healthcare facilities.

My next dose of Spinraza has been postponed. What should I do?
Receiving Spinraza on time is a high priority. But there may be other complicating priorities that result in delays. We would generally recommend that getting doses as close to schedule is important. A few weeks delay should not have a huge impact, as there may be specific personal health status issues or local COVID-19 issues that could influence your timing options. If your scheduled Spinraza dose has been delayed, please work closely with your healthcare provider team to reschedule the Spinraza dose for as soon as reasonably possible. We do not recommend viewing Spinraza doses as elective or non-urgent and dosing should not be delayed for a significant period of time.

My child is scheduled to receive treatment with Zolgensma. Should I proceed?
Receiving Zolgensma is a high priority. Every precaution should be taken to avoid exposure to COVID-19. Some communities have very high rates of COVID-19, resulting in strain on the healthcare system and interfering with being able to receive Zolgensma as planned. Planning to receive Zolgensma should include strategies to avoid possible exposures to COVID-19, such as avoiding waiting rooms and being placed in a procedure room shortly after arrival. Please work with your healthcare provider to schedule administration as soon as possible and ensure safety to receive Zolgensma. We do not recommend viewing Zolgensma treatment as elective or non-urgent and dosing should not be delayed for a significant period of time.

Am I at increased risk for COVID-19 because I had Zolgensma?
While taking prednisone/prednisolone, which can temporarily suppress the immune system, people should be considered at higher risk for serious illness from COVID-19 and should take all recommended precautions very seriously. We are not aware of any specific information regarding COVID-19 among people who have received gene therapy, including Zolgensma.

I am not currently on a treatment, but I am working with my healthcare provider to obtain approval to start treatment. What should I do?
Receiving an FDA-approved treatment for SMA as quickly as possible is a high priority. Continue to work with your healthcare provider to obtain approval and schedule dosing for as soon as reasonably possible. Planning to receive an SMA treatment should include strategies to avoid possible exposures to COVID-19, such as avoiding waiting rooms and being placed in a procedure room shortly after arrival.

Should I continue to have in-home health nursing and other medical professionals in my home?
Safety and avoidance of exposure to infections is paramount. Healthcare professionals in the home are often providing critical services to maintain health, safety, and function. Each person and their family will need to decide whether to continue having these in-home services. When healthcare providers are in the home, ensure they wash hands frequently and have ready access to hand sanitizer and facemasks. In-home healthcare providers should be free of symptoms.

How much should I stock up on supplies and medications?
We recommend having at least two (2) weeks of medications and supplies on hand. This includes supplies for equipment, such as cough machines and other respiratory equipment, formula and feeding supplies, and any other regularly ordered supplies from durable medical equipment companies and suppliers.

What should I do if I am currently participating in a clinical trial?
If you are participating in clinical research, please be in communication with the research team about your study visits. Many studies are making changes that allow you to stay in the study while also making your safety a top priority.

This situation has caused a lot of anxiety in me and my family. What can I do?
Many people with SMA are experiencing anxiety because of all the uncertainty around COVID-19, particularly because of the higher risk among the community. In addition to guidelines for people who are at higher risk, the CDC provides information on coping with the fear and anxiety caused by the COVID-19 outbreak.

How do I get tested for COVID-19?
The CDC recommends that people who have symptoms of COVID-19 be tested. If you have symptoms of COVID-19 (e.g., fever, cough, shortness of breath), contact your doctor. Because the supply of testing kits is currently limited, many areas are only testing people who need to be hospitalized or healthcare workers who become sick. People who have not experienced symptoms, but are worried about getting COVID-19, are unlikely to be tested right away. Your doctor may recommend you be tested, and the CDC recommends doctors work with local and state health departments to coordinate testing through public health laboratories.

Kenneth Hobby
President, Cure SMA

Mary Schroth, M.D.
Chief Medical Officer, Cure SMA

Jill Jarecki, Ph.D.
Chief Scientific Officer, Cure SMA

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

We want to thank everyone who joined us for yesterday''s Coronavirus (COVID-19) Community Webinar: Updates and Next Steps. We hope that the webinar helped answer questions and address concerns regarding the ongoing Coronavirus (COVID-19) public health crisis. During this time, Cure SMA would like to continue to be a resource for the entire SMA community.

If you were not able to join us, a recording of the webinar is now available to view online. 

Please note: All discussions around support and care items are intended for U.S. audiences only.

For more support and questions related to COVID-19, visit www.curesma.org/covid19. 

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

There are many things that make the SMA community special, but one of the most important things we share is our ability to unite and overcome a challenge. Since 1984, we have demonstrated strength in the face of adversity. Last week, this was on full display during our first-ever Virtual SMA Conference. No matter the task, we will continue to come together to further our mission and support everyone affected by SMA.

Today is the perfect time to take action. Neotech Products is matching every gift made to Cure SMA this weekend, up to $2,000. By donating, you can have twice the impact in the SMA community, so make a gift today and double your support!

As a reminder, every donor that makes a gift of $50 or more on our website or through the mail by June 28 will have their name added to our Donor Appreciation Wall. There will be more information in the coming weeks about how we are showing our love for passionate donors like you.

Thank you in advance for your support. 

Very Best,

Erin Kelly 
Vice President of Development, Cure SMA 

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

For the past three weeks, Cure SMA has been making the most of SMA Awareness Month by educating about SMA and demonstrating our continued dedication to the SMA community. Our 2020 Summit of Strength Webinar Series embodies this commitment. These, and other new programs, provide virtual learning opportunities about topics tailored to life with SMA during quarantine, while allowing individuals and families to attend from the safety of their homes.

No matter what challenges lie ahead, we will continue to leverage our determination and innovation to provide the resources that the SMA community needs and depends on. Our goal is to expand these virtual opportunities and, with your support, create interactions and increase connections within the SMA community.

If you make a gift of $25 of more this weekend (August 21-23), you will be entered into a drawing to win a Cure SMA “Swag Bag” sponsored by Natera, a worldwide genetic testing company that offers carrier screening for SMA. Enter by making a donation today. Earn a chance to win Cure SMA merchandise and help provide new resources for all people facing today’s challenges.

Thank you in advance for your support. 

Very Best,

Erin Kelly
Vice President of Development, Cure SMA

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

Cure SMA has worked for 36 years to ensure that no one in our community is ever alone when facing spinal muscular atrophy. The strength of our community has been on full display during the current COVID-19 pandemic.

We have been able to launch many new COVID-19 support programs helping over 3,000 patients and families within just a few weeks. Additionally, all of our ongoing support programs have now been migrated to virtual platforms. You can view our response and read more about these programs by visiting our COVID-19 Information Center.

In addition to rapidly launching these new programs during this difficult time, because of your support Cure SMA has also been able to continue advancing our core research and care programs. We recently funded over $1.1 million in new research grants and have grown our Care Center Network to 20 sites.

Thank you for all you do to keep our community safe and strong. No matter what lies ahead, we remain committed and focused on our mission.

Very Best,

Kenneth Hobby
President, Cure SMA

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

Recordings of the Summit of Strength Virtual Program Series from May 20th and 27th are now available to watch online.

May 27th SMA Nutrition: Now More Than Ever ➱

May 20th Physical Therapy at Home ➱

We hope these webinars offer support and guidance in your everyday life. Keep an eye on Cure SMA’s website for added webinar topics in the upcoming months!  

Thank you to the National Presenting Sponsors for the 2020 Summit of Strength Program: AveXis, Biogen, and Genentech. We would also like to recognize Platinum Sponsor, Scholar Rock, for its support.

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

On Friday, March 27th, at 11:30am CST (9:30am PST/10:30am MST/12:30pm EST) Cure SMA will host an SMA community webinar to help answer questions and address concerns we have heard in recent days and weeks. During this time, Cure SMA would like to continue to be a resource for the entire SMA community.

Register ▶

The webinar will provide the community with up-to-date virus information and Cure SMA activities and programs created in response to the COVID-19 public health crisis. Hear from Cure SMA leadership team, including Kenneth Hobby, President of Cure SMA; Mary Schroth, M.D., Chief Medical Officer; and Colleen McCarthy O''Toole, VP of Events and Family.

The 60-minute webinar will also include a Q&A session based on questions submitted in advance of the call. We encourage you to submit your questions when you register or by emailing webinar@curesma.org, so that we can plan to answer as many as possible. We look forward to you joining us!

This webinar is intended for U.S. audiences only. 

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Dear Friend,

With 2021 less than 48 hours away, we are asking for your help to maintain our momentum as we enter the New Year. The next breakthroughs in spinal muscular atrophy (SMA) care are on the horizon with emerging targeted and combination therapies, more customized support programs, and a standard for care for all people with SMA. We have ambitious goals ahead of us and know that, together, we can create another year of life-changing progress for everyone in the SMA community.

Can we count on your support to help us shape a stronger future for the SMA community?

There is still time to make a gift to Cure SMA that is 100% tax-deductible for the 2020 calendar year. Make a gift today to take advantage of these tax incentives while also showing your support and passion for Cure SMA’s mission.

Thank you in advance for your support. 

Very Best,

Erin Kelly
Vice President, Development 

800-886-1762 | info@curesma.org | curesma.org

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Dear Friend,

While 2020 did not pan out as we had expected, Cure SMA remains extremely optimistic about the future for our community in 2021 and beyond. Here are the reasons why:

Research and Treatment
The research Cure SMA funds continues to expand our knowledge of SMA and deliver treatment opportunities, as reflected in the progress seen with Evrysdi, Spinraza, and Zolgensma. With these three treatments now approved by the FDA, in 2021 we are advancing new research that focuses on non-SMN targeted therapies, symptom and muscle-targeting treatments, and the development of combination therapies that increase strength and enable independence for all people with SMA.

Early Identification of SMA
As of today, 7 in 10 babies born in the U.S. are screened for SMA at birth and we are on track to exceed 90 percent of all births by this time next year. This will lead to early diagnosis and then early treatment, and ultimately better outcomes. In 2021, Cure SMA will continue working with all non-screening states to implement newborn screening for SMA to ensure babies in every state have the best chance at a healthy, full life.

Patient Care
With new information from our SMA Clinical Data Registry, we are improving our understanding of the best care for SMA. By following patient outcomes and treatment progression, we will create an evidence-based standard of care. An important goal in 2021 will be the continued growth and integration of the Cure SMA Care Center Network to ensure a comprehensive registry and improved local patient care.

Advocacy for Treatment and Services
In 2021, Cure SMA will continue its efforts to educate public and private insurers on the ongoing needs of the SMA community and the importance of access to life-altering medications. Additionally, Cure SMA will look to accelerate our work on policy issues most important to adults and young adults with SMA.

Local Support 
As SMA changes, so must our efforts to unite local communities through Cure SMA Chapters, support programs including Summits of Strength, and community events like Walk-n-Rolls. With a greater local presence, we can ensure access to approved treatments and care, and we can create customized programs that meet the new needs of everyone with SMA in local communities.

This new year is a time to set up to reach big goals—and that is what Cure SMA is doing. As always, we thank you for your unwavering support and partnership and look forward to keeping you updated on all our efforts throughout the coming year.

Very best,

Kenneth Hobby,
President, Cure SMA

800-886-1762 | info@curesma.org | curesma.org

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