Research

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

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
Blog Post

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

By Jeanene Swanson | March 7, 2019
Blog Post

Clinical Trial Alert: Amicus Therapeutics Seeks Participants for a Phase 3 Late-Onset Pompe Disease Study

By MDA Staff | March 5, 2019
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