Science + Research

Find the latest science and research updates as well as information to help you understand this ever-evolving field.

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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
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News on LEMS Treatment and Research

By Amy Madsen | March 26, 2019
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Clinical Trial Alert: Researchers at Neurogene Seek Participants for a CMT4J Natural History Study

By MDA Staff | March 21, 2019
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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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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
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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
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MDA Releases New DMD Fact Sheet During Duchenne Muscular Dystrophy Awareness Week

By MDA Staff | February 13, 2019