Industry Updates
New Phase 2 TOPAZ Trial Data Indicate Positive Trends in Quality-of-Life Measures Over 24 Months with Apitegromab for Nonambulatory Patients with Types 2 and 3 SMA
Scholar Rock yesterday announced new quality-of-life (QoL) data from its Phase 2 TOPAZ trial extension period evaluating patient outcomes after 24 months of treatment which […]
Read More ›SMA Community Update from Novartis Gene Therapies
Novartis Gene Therapies has provided an update to the SMA Community. Check out the latest here!
Read More ›SMA Community Update from Novartis Gene Therapies
Novartis Gene Therapies has provided an update to the SMA Community. Check out the latest here!
Read More ›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 […]
Read More ›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.
Read More ›SMA Community Update from Novartis Gene Therapies
We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›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 […]
Read More ›