Drug Development

Blog Post

CMTA and MDA Co-Fund Proof-of-Concept Study Using AAV Vector to Deliver Gene Replacement Therapy in X-linked Charcot-Marie-Tooth Disease

By Jeanene Swanson | March 1, 2019
Blog Post

FDA Accepts Sarepta Therapeutics’ New Drug Application (NDA) for Golodirsen to Treat DMD Amenable to Exon 53 Skipping

By Jeanene Swanson | March 1, 2019
Blog Post

Solid Biosciences Announces Preliminary Results of DMD Gene Therapy Trial

By Jeanene Swanson | February 8, 2019
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Blog Post

Catalyst Pharmaceuticals Announces Positive Results from Phase 2b Trial of Firdapse in Patients with Muscle-Specific Kinase Antibody Positive Myasthenia Gravis (MuSK-MG)

By Jeanene Swanson | January 30, 2019
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Blog Post

Clinical Trial Alert: Brainstorm Cell Therapeutics Seeks Participants for a Phase 3 ALS Study

By MDA Staff | January 16, 2019
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Blog Post

FDA Awards Fast Track Status to RNS60, Revalesio’s Investigational Therapy for ALS

By Jeanene Swanson | January 16, 2019
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Blog Post

Clinical Trial Alert: Orphazyme Seeks Participants for a Phase 3 ALS Study

By MDA Staff | January 11, 2019
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Clinical Trial Alert: Catabasis Seeks Participants for a Phase 3 DMD Study

By MDA Staff | January 10, 2019
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Wave Life Sciences Announces Phase 2/3 Duchenne Muscular Dystrophy Clinical Trial of Suvodirsen (WVE-210201) Selected for FDA Complex Innovative Trial Designs Pilot Program

By Jeanene Swanson | January 8, 2019
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FDA Grants Orphan Drug Designation to Myonexus Therapeutics’ MYO-102 Gene Therapy Drug for Limb-Girdle Muscular Dystrophy Type 2D

By Jeanene Swanson | January 4, 2019