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 »
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
Cure SMA is excited to share an important update to our travel support programs. In response to community feedback and the growing need for accessible travel support for younger individuals, we have expanded eligibility for our Travel Support Package to now include teens with SMA. Previously available only to adults with SMA, the Travel Support
Cure SMA Expands Travel Support Package Eligibility to Include Teens with SMA Read More »
Today, Genentech announced the discontinuation of emugrobart (an investigational anti-myostatin antibody, also known as GYM329) into Phase III development for spinal muscular atrophy (SMA). The decision to stop clinical development of emugrobart in SMA follows a comprehensive assessment of the totality of the evidence from Part 1 of the MANATEE ( NCT05115110 ) study –
Genentech Issues Community Letter Regarding Emugrobart 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
