News

Read the latest news about issues affecting the neuromuscular disease community.

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

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

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

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

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

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

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

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Wisconsin Sen. Tammy Baldwin Introduces Air Carrier Access Amendments Act

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Clinical Trial Alert: ReveraGen BioPharma Seeks Participants for a Phase 2b DMD Study

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