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
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
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 Administration (FDA). This enables the FDA to make patient-centered decisions about new SMA drugs. This is a critical time for patient input on SMA treatment, as new therapies and variations
Cure SMA Shares Patient Voice and Needs with FDA Read More »
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 unmet needs of individuals with SMA and barriers they face in accessing essential healthcare and treatments. Patient and clinical-reported data have chronicled how SMA is rapidly changing due to the
Cure SMA Educates Insurers on Unmet Needs and Access Barriers of the SMA Community Read More »
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 Committee recognized the unmet medical needs of individuals with SMA, including those from military and veteran families, by this year adding “spinal muscular atrophy” within a biomedical research program managed
Key Congressional Committee Recommends New Federal Research in SMA Read More »
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 a time to come together to celebrate progress, honor experiences, reflect on challenges, and build momentum for the future. The theme of SMA awareness month, “Breakthroughs Begin with You,” puts
This August is Spinal Muscular Atrophy (SMA) Awareness Month! Read More »
