Science + Research

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

Blog Post

MDA Partners with the Broad Institute on Rare Genomes Project for Limb-Girdle Muscular Dystrophy

By Jeanene Swanson | April 12, 2019
Blog Post

Audentes Therapeutics Announces Plans to Develop Gene-Targeted Therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1

By Jeanene Swanson | April 11, 2019
Blog Post

Muscular Dystrophy Association Awards Eight Grants Totaling More Than $2 Million for ALS Research

By Jeanene Swanson | April 8, 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
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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
Featured Article

News on LEMS Treatment and Research

By Amy Madsen | March 26, 2019
Blog Post

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
Blog Post

FDA Grants Breakthrough Therapy Designation to Amicus’ Experimental Therapy, AT-GAA, for Late-Onset Pompe Disease

By Jeanene Swanson | March 7, 2019