Front Page 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 […]
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 […]
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 […]
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 […]
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. […]
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 […]
Read More ›Cure SMA Advocacy Highlights of 2024
As 2024 draws to a close, Cure SMA reflects on an extraordinary year of advocacy milestones that have advanced the priorities of individuals with spinal […]
Read More ›Cure SMA Celebrates New DOT Rule for Accessible Air Travel
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 […]
Read More ›Biohaven Provides Update on Taldefgrobep Alfa Development Program for Spinal Muscular Atrophy
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 […]
Read More ›SMA Community Advocacy Wins and Champions Celebrated at Cure SMA Event
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. […]
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