Industry Updates
Community Statement from Novartis Gene Therapies: Zolgensma Safety Monitoring Update on Acute Liver Failure
Dear SMA Community, Novartis Gene Therapies is committed to patient safety and the ongoing monitoring of adverse events as it relates to the use of […]
Biogen Shares Community Statement for SMA Awareness Month
Learn about Biogen’s latest news in SMA by viewing their Community Statement (available as a PDF) and a related video.
SMA Community Update from Novartis Gene Therapies
We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.
FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy
Genentech, a member of the Roche Group, today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to […]
Biohaven Licenses a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA)
Biohaven Pharmaceutical Holding Company Ltd., announced today that it entered into a worldwide license agreement with Bristol Myers Squibb for the development and commercialization rights to […]
Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA
Biogen Inc. and Ionis Pharmaceuticals, Inc., recently announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license […]
Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With SMA
Genentech, a member of the Roche Group, recently announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new […]
Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial
Dear SMA Community, We are excited to announce the design of SAPPHIRE, a Phase 3 clinical trial to further evaluate the safety and efficacy of […]
Community Update from Novartis Gene Therapies
We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.
Update on Genentech/Roche Initiation of MANATEE Clinical Study
We are pleased to share an update on Genentech’s, a member of the Roche Group, initiation of MANATEE, a new global Phase 2/3 clinical study […]