Clinical Trials

Blog Post

Clinical Trial Alert: PTC Therapeutics Seeks Participants for a Phase 3 DMD Study

By MDA Staff | April 24, 2019
Blog Post

Sarepta Therapeutics Announces Positive Interim Results from Study of Casimersen to Treat DMD Amenable to Exon 45 Skipping

By Jeanene Swanson | April 4, 2019
Blog Post

FDA Grants Orphan Drug Designation to Acceleron Pharma’s ACE-083 Muscle Growth Drug for Charcot-Marie-Tooth Disease

By Jeanene Swanson | April 3, 2019
Blog Post

Clinical Trial Alert: Researchers at Neurogene Seek Participants for a CMT4J Natural History Study

By MDA Staff | March 21, 2019
Blog Post

Sarepta Therapeutics Announces Positive Interim Results in Gene Therapy Trial for LGMD2E and Acquisition of Myonexus Therapeutics

By Jeanene Swanson | March 15, 2019
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Clinical Trial Alert: ReveraGen BioPharma Seeks Participants for a Phase 2b DMD Study

By MDA Staff | March 12, 2019
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FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease

By Jeanene Swanson | March 7, 2019
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Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study

By MDA Staff | March 5, 2019
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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

Long-Term Results of Extension Study Validate Surgery as a Treatment for Myasthenia Gravis

By Jeanene Swanson | February 12, 2019