Latest News
SMA Community Risk Tolerance Update: Comparison of 2022 and 2017 SMA Risk/Benefit Survey Data
One of Cure SMA’s top priorities is to relay the SMA community’s treatment experiences and preferences to the United States Food and Drug Administration (FDA). This helps the FDA make […]
Read More ›FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA)
“Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval marks another significant step forward,” […]
Read More ›Cure SMA Launches Advocacy Campaign to Secure New Federal Research for SMA
To address the chronic health needs of individuals with spinal muscular atrophy (SMA), Cure SMA and the SMA community have launched a new advocacy campaign in support of federal research […]
Read More ›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 […]
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