Industry Updates

Cure SMA Update on SMA Treatment Delays

September 24, 2025
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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

September 23, 2025
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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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Scholar Rock Receives News on Biologics License Application (BLA) for Apitegromab from the U.S. FDA

September 23, 2025
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Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has delayed the approval with a Complete Response Letter (CRL) for the apitegromab […]

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

June 25, 2025
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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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Novartis Gene Therapies Releases Spring SMA Community Update Letter

May 5, 2025
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Novartis Gene Therapies recently released an update on their continued collaboration with the SMA community through events, advisory boards, clinical insights, and ongoing efforts to […]

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FDA Grants Priority Review for Biologics License Application (BLA) for Apitegromab as a Treatment for Spinal Muscular Atrophy

March 25, 2025
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Today, Scholar Rock announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for apitegromab, an investigational muscle-targeted treatment […]

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FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA)

February 12, 2025
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  “Evrysdi has robust potential to modify the SMA disease trajectory, and has already been used to treat thousands of patients to date. This approval […]

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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)

January 29, 2025
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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 […]

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

January 23, 2025
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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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Novartis Intrathecal Gene Therapy Onasemnogene Abeparvovec Phase III Study Meets Primary Endpoint in Children and Young Adults with SMA

December 30, 2024
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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 […]

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