April 2021

Community Spotlight: Elizabeth Lockwood 

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In honor of National Volunteer Month in April, Cure SMA has shared stories and quotes from volunteers who support our programs and further grow our community. Cure SMA is incredibly grateful for the  volunteers who keep this organization strong.   Elizabeth Lockwood is a grandmother. Her grandchildren are Emma and Nick, both young adults with spinal muscular atrophy (SMA). Like most in the community, Elizabeth had […]

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Cure SMA-Funded Researchers at Columbia University Publish Paper on Fatigue in SMA

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Dr. Jacqueline Montes and colleagues at Columbia University—part of the Pediatric Neuromuscular Clinical Research Network (PNCRN) funded by Cure SMA—have published a paper in the journal Annals of Clinical and Translational Neurology, titled “Diminished muscle oxygen uptake and fatigue in spinal muscular atrophy.” This project, funded through the PNCRN, was an observational study evaluating muscle oxygen update

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Community Statement from Novartis Gene Therapies

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Dear SMA Community, Novartis Gene Therapies is committed to working with our partners in the SMA community to make a difference in the diagnosis, treatment, and care of those affected by SMA. We would like to announce that we will initiate SMART, a Phase 3b clinical study to further evaluate the safety and efficacy of

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Cure SMA Attends Spring 2021 Professional Organization Conferences to Promote Findings from Industry Collaboration Initiatives

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Cure SMA is pleased to announce its participation in the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference (March 15-18, 2021) and the American Academy of Neurology (AAN) 2021 Virtual Annual Meeting (April 17-22, 2021). Poster presentations at these meetings feature data from research initiatives launched by the Cure SMA Industry Collaboration (SMA-IC).

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Genentech Shares New Evrysdi Data on Improved Motor Function and Survival in Babies with SMA Type 1

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Genentech, a member of the Roche Group, has shared new 2-year data from Part 2 of FIREFISH, a Phase 2/3 global study evaluating Evrysdi (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type 1 SMA. The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to

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Biogen Shares New Spinraza Data Exploring Opportunities to Improve Outcomes for SMA Patients

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Biogen has shared new data from the Spinraza (nusinersen) clinical development program aimed at optimizing outcomes for people with SMA and advancing understanding of the disease. Exploring Opportunities to Optimize Treatment in SMA Building on the proven efficacy and well-established safety of Spinraza in a broad range of patients with SMA, the Phase 2/3 DEVOTE study is evaluating the

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Participate in Annual Cure SMA Community Update Survey – Open Now!

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Last year presented the us with unique challenges, some we are still managing today. However, it has also reinforced the importance of understanding what the SMA community is thinking, feeling, and experiencing in their day-to-day. Knowing our community’s needs allows us to better serve you—whether it be advocating for comprehensive research, educating regulators, payers, and

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Cure SMA Presents Data on Cure SMA Care Center Network and SMA Clinical Data Registry

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Cure SMA’s primary focus is supporting care of the highest value to individuals with SMA. As a result of treatments approved by the U.S. Food and Drug Administration (FDA), as well as successful initiatives like the Cure SMA Care Center Network and SMA Clinical Data Registry, we have made significant progress enhancing the lives of

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Cure SMA Launches LifeVac Support Program to Help in Choking Event

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Along with funding SMA research and care, Cure SMA is proud to provide thousands of individuals with SMA and their families with vital support they need to navigate life with SMA. In an effort to always be offering additional resources, we are thrilled to launch a new program for all individuals with SMA to help

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Scholar Rock Community Statement on TOPAZ 12-Month Topline Data

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Scholar Rock provided the below community statement on data for apitegromab (SRK-015) following the distribution of this company press release.  Dear Members of the SMA Community, We are pleased to share 12-month topline results from our TOPAZ study, a Phase 2 clinical trial to evaluate the safety and efficacy of apitegromab (SRK-015) in individuals with

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