Industry Updates
Cure SMA Shares Updates on the Phase 10 SMA Industry Collaboration
Earlier this year, Cure SMA launched an expanded Phase 10 of the SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together […]
Novartis Receives FDA Approval of Itvisma for the Treatment of SMA
Novartis today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for Itvisma (onasemnogene abeparvovec-brve) to treat adults and children 2 years of age and older with […]
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 […]
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 […]
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, […]
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 […]
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 […]
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 […]
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 […]
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 […]