Novartis Intrathecal Gene Therapy Onasemnogene Abeparvovec Phase III Study Meets Primary Endpoint in Children and Young Adults with SMA

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Novartis today announced positive topline results from the Phase III STEER study. This pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to less than 18 years who are able to sit but have never walked independently. Efficacy and […]

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Cure SMA Celebrates New DOT Rule for Accessible Air Travel

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Cure SMA is thrilled to share a major win for the SMA community: the U.S. Department of Transportation (DOT) has finalized its rule on ensuring safe accommodations for air travelers with disabilities using wheelchairs. This significant advancement underscores the importance of equity in air travel and represents a critical step toward making flights safer and

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Biohaven Provides Update on Taldefgrobep Alfa Development Program for Spinal Muscular Atrophy

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In the RESILIENT SMA study, Biohaven announced today that taldefgrobep alpha showed clinically meaningful improvements in motor function at all time points on the Motor Function Measurement-32 scale (MFM-32), but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care (SOC) group. Biohaven plans to

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SMA Community Advocacy Wins and Champions Celebrated at Cure SMA Event

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Individuals with spinal muscular atrophy (SMA) and their families recognized congressional champions and celebrated advocacy progress at Cure SMA’s Hope on the Hill event. U.S. Representative Sam Graves (MO-6) Photo: Jay Mallin U.S. Representative Pete Stauber (MN-8) Photo: Jay Mallin U.S. Representative Mark DeSaulnier (CA-10) Photo: Jay Mallin This year’s event, which was held on

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Celebrate 40 Years of Impact – Join Us in Making a Difference!

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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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Spinal Muscular Atrophy (SMA) Update in Best Practices: Recommendations for Treatment Considerations

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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

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Make an Impact with Cure SMA’s Mission, Milestones, Momentum Campaign

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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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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,

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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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