Thank you for being an essential part of this journey and for believing in our mission. As we mark 40 years of dedicated service to the spinal muscular atrophy (SMA) community, we reflect on our journey and the incredible milestones we’ve achieved together. In 1984, a small group of parents founded Families of Spinal Muscular […]
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As part of our ongoing efforts to drive quality health care standardization and improvements for the SMA community, Cure SMA is pleased to announce the next publication in a series of resources designed to support healthcare professionals, and individuals with SMA, and their caregivers. “Spinal Muscular Atrophy (SMA) Update in Best Practice: Recommendations for Treatment
We’re kicking off our Mission, Milestones, Momentum Year-End Campaign with an incredible $50,000 matching gift. In honor of Hunter Rhodes, living with SMA, the Johns Family has generously donated $50,000 and has challenged the SMA community to double their impact by joining them in making a gift to Cure SMA today–no matter how big or
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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,
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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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
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
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
This week, Cure SMA and advocates with SMA celebrated Disability Pride and the 34th Anniversary of the Americans with Disabilities Act (ADA) with a special reception at the White House. The event marked a significant occasion to reflect on the progress made and the challenges that remain in achieving full equality and inclusion for people
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The positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA. Biogen plans to submit for regulatory approval of this investigational dose regimen. Biogen today announced positive, topline data from the pivotal cohort (Part B) of the Phase 2/3 DEVOTE study evaluating the safety and efficacy of a
