The 10th Annual Cure SMA Community Update Survey Is Now Available

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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 […]

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Breakthroughs Built Together: Help Cure SMA Close a Critical Research Funding Gap

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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

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Cure SMA Launches Phase 11 SMA Industry Collaboration

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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

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Scholar Rock Resubmits Biologics License Application (BLA) for Apitegromab to the FDA

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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

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Biogen Receives FDA Approval of High Dose SPINRAZA for the Treatment of SMA

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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

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Cure SMA Awards $150,000 Grant to Tetsuya Akiyama, MD, PhD, Stanford University School of Medicine

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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

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Cure SMA Expands Travel Support Package Eligibility to Include Teens with SMA

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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

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Genentech Issues Community Letter Regarding Emugrobart

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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 –

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Cure SMA Awards $75,000 Grant to Lyndsay Murray, PhD, University of Edinburgh in Scotland

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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

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Evrysdi Risdiplam Tablet Is Now Approved For Broader Use In SMA

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We’re excited to share an important update for the spinal muscular atrophy (SMA) community. The U.S. Food and Drug Administration (FDA) has approved a label expansion for Evrysdi® (risdiplam) tablets, effective February 10, 2026. The tablet formulation is now approved for administration via nasogastric (NG) or gastrostomy (G) feeding tubes after dispersion in water. This

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