Front Page News
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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›Cure SMA Shares Patient Voice and Needs with FDA
One of Cure SMA’s top priorities is to relay the SMA community’s experiences, preferences, and unmet needs to the United States Food and Drug […]
Read More ›Cure SMA Educates Insurers on Unmet Needs and Access Barriers of the SMA Community
Cure SMA completed a year-long advocacy effort to educate public and private insurers about the current state of spinal muscular atrophy (SMA), including the […]
Read More ›Key Congressional Committee Recommends New Federal Research in SMA
A key congressional committee approved fiscal year 2026 legislation that now prioritizes new federal research funding for spinal muscular atrophy (SMA). The U.S. Senate Appropriations […]
Read More ›This August is Spinal Muscular Atrophy (SMA) Awareness Month!
Whether you live with SMA, know someone who does, work in research or care, or are beginning your journey with the community-SMA Awareness Month is […]
Read More ›