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

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Muscular Dystrophy Association Bridges Clinical and Academic Neuromuscular Research for the First Time at Annual Meeting

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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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News on LEMS Treatment and Research

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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