Latest 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 Biogen’s high-dose nusinersen/Spinraza. While there […]
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 Application (sNDA) for the high-dose […]
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 Biologics License Application (BLA) for […]
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 key priorities of the SMA […]
Read More ›2025 Hope on the Hill Celebrates Day of Advocacy in Washington, DC
Cure SMA celebrated a day of advocacy on Capitol Hill on September 16 with more than 170 individuals with spinal muscular atrophy (SMA), their families, and SMA community partners at […]
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 Care Meeting. Their common goals […]
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