Cure SMA is pleased to announce the publication of the 5th annual State of SMA report. Drawing on data collected by Cure SMA through 2025, this report features insights into the progress and ongoing challenges impacting people with spinal muscular atrophy (SMA) and their families. Previously, the annual report has also been used to advocate […]
Cure SMA Releases the 2025 Annual State of SMA Report Read More »
Today, Scholar Rock shared that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational treatment for spinal muscular atrophy (SMA), with a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2026. This is a meaningful step forward in the path toward a potential new
Cure SMA has released a new policy brief highlighting the essential role Medicaid-funded home care plays in the lives of individuals with spinal muscular atrophy (SMA), a rare neuromuscular disease. Due to severe muscle weakness, many individuals with SMA rely on Medicaid-funded home care to meet their complex daily needs, including eating, bathing, bathroom support,
