Cure SMA Launches Advocacy Campaign to Secure New Federal Research for SMA

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To address the chronic health needs of individuals with spinal muscular atrophy (SMA), Cure SMA and the SMA community have launched a new advocacy campaign in support of federal research for SMA. Through the campaign, Cure SMA seeks dedicated SMA research funding through a federal research program managed by the U.S. Department of Defense (DOD). […]

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Rare Disease Month 2025: Driving Progress Through Research

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February marks Rare Disease Month, a time to amplify the voices of the 300 million people worldwide living with a rare disease—including the individuals in the U.S. impacted by spinal muscular atrophy (SMA). Throughout this month, and especially during Rare Disease Week (starting February 23rd), we’re committed to advancing research, raising awareness, and advocating for

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Scholar Rock Submits Biologics License Application (BLA) to the U.S. FDA for Apitegromab as a Treatment for Patients with Spinal Muscular Atrophy (SMA)

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  “With the strength of our Phase 3 data as the foundation of our submission, we look forward to continuing to work closely with the FDA through the review of our BLA on behalf of patients and families living with SMA.” Today, Scholar Rock announced the submission of a Biologics License Application (BLA)  to the

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Biogen Announces that FDA and EMA Accept Applications for Higher Dose Regimen of Nusinersen in SMA

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“We are committed to supporting individuals with SMA and their families by advancing research that aims to answer critical questions for the community.” Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) and the European Medicines Agency (EMA) has validated the application for a

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All 2025 Walk-n-Roll Events Are Open

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Cure SMA warmly invites you to join us at one of our 2025 Walk-n-Roll events—where community meets impact! This year, for the first time, you can register and start fundraising for both spring and fall events right now. Walk-n-Roll isn’t just a fundraiser; it’s a celebration of progress, hope, and togetherness. It’s a chance to

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Cure SMA Caregiving Priority Signed into Law

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On January 4th, 2025, President Biden signed the Think Differently Database Act (H.R. 670) into law, marking a significant legislative victory for the SMA community. This law will create an interactive, searchable webpage providing federal, state, and local information about caregiving services, including Medicaid Home and Community-Based Services (HCBS) eligibility, to individuals with disabilities and

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Novartis Intrathecal Gene Therapy Onasemnogene Abeparvovec Phase III Study Meets Primary Endpoint in Children and Young Adults with SMA

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Novartis today announced positive topline results from the Phase III STEER study. This pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to less than 18 years who are able to sit but have never walked independently. Efficacy and

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Cure SMA Celebrates New DOT Rule for Accessible Air Travel

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Cure SMA is thrilled to share a major win for the SMA community: the U.S. Department of Transportation (DOT) has finalized its rule on ensuring safe accommodations for air travelers with disabilities using wheelchairs. This significant advancement underscores the importance of equity in air travel and represents a critical step toward making flights safer and

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Biohaven Provides Update on Taldefgrobep Alfa Development Program for Spinal Muscular Atrophy

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In the RESILIENT SMA study, Biohaven announced today that taldefgrobep alpha showed clinically meaningful improvements in motor function at all time points on the Motor Function Measurement-32 scale (MFM-32), but the treatment arm did not statistically separate on the primary outcome at Week 48 compared to the placebo+standard of care (SOC) group. Biohaven plans to

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