Author name: Sarah McCall

Administration Officials & Members of Congress Highlight SMA Community Advocacy at 12th Annual Hope on the Hill Washington, D.C. Event 

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Two high-ranking Administration officials and several Members of Congress from key congressional committees participated in Cure SMA’s November 29th Hope on the Hill dinner and reception, an annual Washington, D.C. event that raises awareness about spinal muscular atrophy (SMA) with key government officials. The 2023 event highlighted key SMA community advocacy priorities, including accessible air […]

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Sweet Success: Baking a Difference Returns for a Second Year

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Breaking and Baking News!   Get ready to dust off those aprons and preheat those ovens because Baking a Difference is back and better than ever for its second year! Created by kids, for kids, with hearts as sweet as the treats they bake, this heartwarming campaign is all set to spread joy and make

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Genentech Shares 1-Year Primary Analysis from RAINBOWFISH

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Last week, Genentech, a member of the Roche Group,  presented positive results from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic SMA, aged from birth to 6 weeks. These data were presented at the 28th World Muscle Society (WMS) Congress. Read more about

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Identifying Biomarkers of Spinal Muscular Atrophy for Further Development

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An important part of Cure SMA’s core mission is to carry out and support research that will improve the quality of life for all people with SMA. We recently published a new research article, “Identifying Biomarkers of Spinal Muscular Atrophy for Further Development.” This article appeared in the July 12, 2023 issue of the peer-reviewed

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NMD Pharma Initiates Phase II Trial for SMA

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NMD Pharma, a clinical-stage biotech company leading the development of novel first-in-class therapies for severe neuromuscular disorders, recently announced that the first SMA patient has been dosed in their Phase II clinical trial of the CIC-1 inhibitor NMD670. To read the full press release, visit this link. MD670 is a first-in-class small molecule inhibitor of

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Scholar Rock Announces Completion of Enrollment for the Phase 3 SAPPHIRE Trial

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Today, Scholar Rock announced that enrollment in their Phase 3 SAPPHIRE trial is now complete. The Phase 3 trial will evaluate the safety and efficacy of apitegromab in nonambulatory patients with Types 2 and 3 spinal muscular atrophy (SMA) who are receiving SMN therapy (either nusinersen or risdiplam). Scholar Rock also announced that it plans to

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