Latest News
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 […]
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 muscular atrophy (SMA) and their […]
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 safe accommodations for air travelers […]
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 Function Measurement-32 scale (MFM-32), but […]
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. Representative Sam Graves (MO-6) Photo: […]
Read More ›