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 are many unknowns regarding timelines and next steps, the most encouraging news in all of this is that the issues named by the FDA are
Cure SMA Update on SMA Treatment Delays Read More »
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 regimen of nusinersen (SPINRAZA) for people living with spinal muscular atrophy (SMA). The CRL requests updates to the technical information included in the Chemistry Manufacturing
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 the treatment of people living with spinal muscular atrophy (SMA). The CRL is related to observations identified during a routine inspection of Catalent Indiana LLC,
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 community. Advocates from 34 states met in-person with 155 congressional offices in Washington, DC, including 87 U.S. House offices and 68 U.S. Senate offices, to
Cure SMA 2025 Hill Day: 24 Hours of Advocacy Impact Read More »
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 the 2025 Hope on the Hill reception. Founded in 2011, Hope on the Hill was created to raise awareness of SMA in Washington, DC, and
2025 Hope on the Hill Celebrates Day of Advocacy in Washington, DC Read More »
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 were to accelerate the pace of research into SMA treatments and to achieve excellence in SMA clinical care. The annual meeting provides an opportunity for
