Industry Updates

FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy

May 31, 2022
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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 […]

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Biohaven Licenses a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA)

February 25, 2022
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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 […]

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Biogen Exercises Option with Ionis to Develop and Commercialize Investigational ASO for SMA

January 26, 2022
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      Biogen Inc. and Ionis Pharmaceuticals, Inc., recently announced that Biogen exercised its option to obtain from Ionis a worldwide, exclusive, royalty-bearing license […]

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Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With SMA

January 25, 2022
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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 […]

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Scholar Rock Shares SMA Community Letter and Announces Design of Phase 3 SAPPHIRE Clinical Trial

November 30, 2021
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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 […]

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Community Update from Novartis Gene Therapies

October 21, 2021
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We are pleased to share a community update on Novartis Gene Therapies’ Clinical Trial Program.

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Update on Genentech/Roche Initiation of MANATEE Clinical Study

October 20, 2021
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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 […]

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New Data Presented at World Muscle Society 2021 Virtual Congress

September 24, 2021
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This week, new data was presented at the World Muscle Society 2021 Virtual Congress. Check it out below!     Scholar Rock presents two posters […]

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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)

September 16, 2021
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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 […]

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Novartis Announces Lift of Partial Clinical Trial Hold, Plans to Initiate a New Study of Intrathecal Zolgensma in Older Patients with SMA

August 3, 2021
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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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