October 2024

Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate

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Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before 6 weeks of age, with most achieving motor milestones similar to children without SMA All children were able to swallow and feed orally, with none requiring permanent ventilation Evrysdi is the only non-invasive SMA therapy and is approved in over 100 countries, […]

Majority of Children with SMA Treated with Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate Read More »

Cure SMA Hosting Puerto Rico Summit of Strength & Walk-N-Roll

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Cure SMA is thrilled to announce the 2025 San Juan, Puerto Rico Summit of Strength,taking place on Saturday, February 1. The Summit of Strength is a community educational program hosted by Cure SMA. These in-person events provide attendees the opportunity to learn from an array of national and local experts surrounding topics that are valuable to

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Cure SMA is Thrilled to Launch New Travel Resources Webpage and a New Travel Support Package

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Along with funding SMA research and care, Cure SMA provides thousands of individuals and families with vital support and resources that help people navigate life with SMA.  Travel should be accessible for everyone but we know that is not often the case. With this in mind, Cure SMA is thrilled to launch some new travel

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New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA

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Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in both individuals previously treated and treatment-naïve to nusinersen Investigational regimen also shows more rapid slowing of neurodegeneration, as measured by neurofilament Biogen plans to submit regulatory applications around the world

New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA Read More »

Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA)

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Today, Scholar Rock announced plans to submit a U.S. Biologics License Application (BLA) and a European Union marketing authorisation application (MAA) in Q1 2025. Scholar Rock, a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today

Scholar Rock Announces Apitegromab Meets Primary Endpoint in Phase 3 SAPPHIRE Study in Patients with Spinal Muscular Atrophy (SMA) Read More »

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