Industry Updates
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 ›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 […]
Read More ›Community Update from Novartis Gene Therapies
We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.
Read More ›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 […]
Read More ›New Data Presented at World Muscle Society 2021 Virtual Congress
This week, new data was presented at the World Muscle Society 2021 Virtual Congress. Check it out below! Scholar Rock presents two posters […]
Read More ›Community Statement from Biogen: New Clinical Study Planned to Evaluate the Potential Benefit of an Investigational Higher Dose of Nusinersen in People with SMA Previously Treated with Evrysdi® (risdiplam)
Biogen is committed to improving health outcomes for people impacted by spinal muscular atrophy (SMA) and generating data to assess if there are treatment pathways […]
Read More ›Novartis Announces Lift of Partial Clinical Trial Hold, Plans to Initiate a New Study of Intrathecal Zolgensma in Older Patients with SMA
Novartis announced that the U.S. Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may […]
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