Front Page News
Scholar Rock Releases SMA Community Update Letter on Apitegromab
Yesterday, Scholar Rock shared an SMA community update letter on their Biologics License Application (BLA) for apitegromab in SMA. As shared on September 23, 2025, […]
Biogen to Initiate Phase 3 Pivotal Study Evaluating Investigational Drug Salanersen
Today, Biogen announced plans to initiate Phase 3 development to evaluate the effectiveness and safety of salanersen, an investigational drug for the treatment of spinal […]
Cure SMA Updates Health Insurance Road Map to Support Patients and Families
Cure SMA has updated its Health Insurance Road Map to help patients, families, and providers navigate the complexities of health insurance. The updated resource reflects […]
Cure SMA and the Luke 18:1 Foundation Launch New Fold & Go Wheelchair Grant Program
Cure SMA is excited to announce the launch of a brand-new equipment grant program, made possible through the generous support of the Luke 18:1 Foundation. […]
Cure SMA Update on SMA Treatment Delays
Dear SMA Community, Yesterday was a disappointing and frustrating day for our community. We learned of two FDA approval delays for Scholar Rock’s apitegromab and […]
Biogen Receives News on Supplemental New Drug Application (sNDA) for High Dose Nusinersen from the U.S. FDA
Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the supplemental New Drug […]
Scholar Rock Receives News on Biologics License Application (BLA) for Apitegromab from the U.S. FDA
Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the apitegromab […]
Cure SMA 2025 Hill Day: 24 Hours of Advocacy Impact
On Tuesday, September 16, about 150 adults with spinal muscular atrophy (SMA) and family members of children with SMA visited Capitol Hill to advocate for […]
SMA Researchers and Clinicians from Around the World Gather at the 29th Annual SMA Research & Clinical Care Meeting (2 of 2)
At the end of June, SMA researchers and clinicians from around the world gathered in Anaheim, California, for the 29th Annual SMA Research & Clinical […]
SMA Researchers and Clinicians from Around the World Gather at the 29th Annual SMA Research & Clinical Care Meeting (1 of 2)
At the end of June, SMA researchers and clinicians from around the world gathered in Anaheim, California, for the 29th Annual SMA Research & Clinical […]