Latest News
Rare Disease Month 2025: Driving Progress Through Research
February marks Rare Disease Month, a time to amplify the voices of the 300 million people worldwide living with a rare disease-including the individuals in the U.S. impacted by spinal […]
Read More ›Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA)
“With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of […]
Read More ›Biogen Announces that FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA
“We are committed to supporting individuals with SMA and their families by advancing research that aims to answer critical questions for the community.” Today, Biogen announced that the U.S. Food […]
Read More ›All 2025 Walk-n-Roll Events Are Open
Cure SMA warmly invites you to join us at one of our 2025 Walk-n-Roll events-where community meets impact! This year, for the first time, you can register and start fundraising […]
Read More ›Cure SMA Caregiving Priority Signed into Law
On January 4th, 2025, President Biden signed the Think Differently Database Act (H.R. 670) into law, marking a significant legislative victory for the SMA community. This law will create an […]
Read More ›Novartis Intrathecal Gene Therapy Onasemnogene Abeparvovec Phase III Study Meets Primary Endpoint in Children and Young Adults with SMA
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 […]
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