Cure SMA is pleased to announce the participation of our Research Department staff in the following conferences: 2026 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference (March 8 – 11, 2026; Orlando, FL) 5th International Scientific Congress on SMA (March 11 – 14, 2026; Budapest, Hungary) 2026 American Academy of Neurology (AAN) Annual Meeting […]
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 »
(opens in new tab) 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
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,
Since 2017, 3,200 people have shared their experiences and unmet needs through the Cure SMA Community Update Survey. The Community Update Survey is an annual online questionnaire Cure SMA uses to collect perspectives and experiences of the SMA community over time. Every individual with SMA and their families bring a unique perspective that, collectively, help
The 10th Annual Cure SMA Community Update Survey Is Now Available Read More »
This spring, we’re asking our community to help us invest $1 million in basic research by June 30. Every breakthrough in spinal muscular atrophy (SMA) research has one thing in common. It wasn’t built by one person or one gift. It was built by people who believed progress was possible. This spring, we’re asking you to help build the next breakthrough
Breakthroughs Built Together: Help Cure SMA Close a Critical Research Funding Gap Read More »
Cure SMA is pleased to announce the launch of Phase 11 initiates for the SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, SMA Europe, Cure SMA, and other nonprofit organizations to share information, ideas, and data to benefit the broader SMA community. Through the Industry Collaboration, we
Cure SMA Launches Phase 11 SMA Industry Collaboration Read More »
(opens in new tab) Today, Scholar Rock announced that it has resubmitted its Biologics License Application (BLA) to the Food and Drug Administration (FDA) for apitegromab, an investigational therapy for the treatment of people living with spinal muscular atrophy (SMA). Apitegromab is an anti-myostatin muscle-targeted treatment. The resubmission follows a previous Complete Response Letter (CRL)
Scholar Rock Resubmits Biologics License Application (BLA) for Apitegromab to the FDA Read More »
(opens in new tab) Biogen today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for the High Dose regimen of SPINRAZA (nusinersen) to treat people living with spinal muscular atrophy (SMA). With broad label approval for all those living with SMA, this is an excellent result for the SMA community. SPINRAZA (nusinersen)
Biogen Receives FDA Approval of High Dose SPINRAZA for the Treatment of SMA Read More »
Each year, Cure SMA invites scientists from around the world to submit funding proposals for basic research projects that address specific unanswered questions in spinal muscular atrophy (SMA) biology. Our Scientific Advisory Board ranks the submitted proposals based on their scientific merit and their alignment with Cure SMA’s research priorities. Funding is then awarded to
