Industry Updates
Scholar Rock and Cure SMA Share Update on Apitegromab and FDA Meeting
Read the Scholar Rock community statement here. Scholar Rock recently held an in-person Type A meeting with the FDA on November 12 to discuss […]
Read More ›Novartis Releases Fall SMA Community Update Letter
Novartis recently released an update on their continued collaboration with the SMA community through fundraising events and community support programs, and they will continue to […]
Read More ›Scholar Rock Releases SMA Community Update Letter on Apitegromab
Yesterday, Scholar Rock shared an SMA community update letter on their Biologics License Application (BLA) for apitegromab in SMA. As shared on September 23, 2025, […]
Read More ›Biogen to Initiate Phase 3 Pivotal Study Evaluating Investigational Drug Salanersen
Today, Biogen announced plans to initiate Phase 3 development to evaluate the effectiveness and safety of salanersen, an investigational drug for the treatment of spinal […]
Read More ›Cure SMA Update on SMA Treatment Delays
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 […]
Read More ›Biogen Receives News on Supplemental New Drug Application (sNDA) for High Dose Nusinersen from the U.S. FDA
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 […]
Read More ›Scholar Rock Receives News on Biologics License Application (BLA) for Apitegromab from the U.S. FDA
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 […]
Read More ›Biogen Announces Advancement of Investigational SMA Drug Salanersen to Registrational Trials Based on Positive Phase 1 Results
“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 […]
Read More ›Novartis Gene Therapies Releases Spring SMA Community Update Letter
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 […]
Read More ›FDA Grants Priority Review for Biologics License Application (BLA) for Apitegromab as a Treatment for Spinal Muscular Atrophy
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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