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SMA Treatment Delays FAQ

Make today a breakthrough. Last Updated: 10/01/25   We know our community has many pressing questions related to the FDA approval delays of Scholar Rock’s […]

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Cure SMA Update on SMA Treatment Delays

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Dear SMA Community, Yesterday was a disappointing and frustrating day for our community. We learned of two FDA approval delays for Scholar Rock’s apitegromab and […]

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Biogen Receives News on Supplemental New Drug Application (sNDA) for High Dose Nusinersen from the U.S. FDA

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Today, Biogen announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the supplemental New Drug […]

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Biogen Announces Advancement of Investigational SMA Drug Salanersen to Registrational Trials Based on Positive Phase 1 Results

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“To see a child dosed with gene therapy at age one and still unable to sit at age five then gain that ability within three […]

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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 […]

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New Higher Dose Nusinersen Efficacy and Safety Data Presented at World Muscle Society Congress, Highlight Potential to Maximize Benefit of Nusinersen in SMA

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Findings from Part B and Part C of the DEVOTE study support the clinical benefits of a higher dose regimen of nusinersen (50/28 mg) in […]

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Biogen Announces Positive Topline Results from Study of Higher Dose Regimen of Nusinersen, Showing Significant Benefit in Treatment of SMA

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The positive study demonstrates the potential for investigational higher dose nusinersen regimen to advance the treatment of SMA.  Biogen plans to submit for regulatory approval […]

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Congressional Action Recognizes Unmet Needs of SMA Community

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This week, a key congressional committee recognized the unmet, everyday living needs of individuals with spinal muscular atrophy (SMA) by urging the National Institutes of […]

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Biogen Releases SMA Community Letter

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On April 19, 2024, Biogen released an update to the SMA community sharing recent data presentations from Spring conferences and latest updates from their SPINRAZA® […]

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Our History

Make today a breakthrough. Cure SMA was founded in 1984 to support the SMA community and fund research to find a cure. In our 40-year […]

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