Biogen recently announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to salanersen, an investigational therapy being studied for the treatment of spinal muscular atrophy (SMA). This designation is intended to help speed the development and review of drugs that show early promise in treating serious conditions. Salanersen is an […]
The Family-Friendly Poster Session is one of the most anticipated events at the Annual SMA Conference! This year’s session will be held on Friday, June 26, from 6:30 pm to 8:30 pm in the Grand Harbor Ballroom at Disney’s Yacht and Beach Club Resort. The Family Friendly Poster Session is an opportunity for families to
Meet the Scientists Behind SMA Research and Discover What’s Coming Next Read More »
As more SMA treatments become available, people living with SMA, their families, and caregivers are asking new questions about how treatments work, whether using more than one treatment could help, and what to consider when thinking about combination therapy. Understanding how SMA treatments work can help individuals and families feel more informed about their options
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
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,
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 »
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 »
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) issued by the FDA
Scholar Rock Resubmits Biologics License Application (BLA) for Apitegromab to the FDA Read More »
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) is an antisense oligonucleotide
Biogen Receives FDA Approval of High Dose SPINRAZA for the Treatment of SMA Read More »
