Drug Development

Mitochondrion in cell cross section
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Simply Stated: Research Updates in Leigh Syndrome

By Sujatha Gurunathan | December 6, 2023
Graphic of the inside of a human cell
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Q&A: Understanding Metabolic Myopathies

By MDA Staff | November 17, 2023
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Clinical Trial Alert: Phase 3 Study of Brepocitinib in Adults with Dermatomyositis

By Sujatha Gurunathan | November 16, 2023
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AGAMREE ® (Vamorolone), Now FDA-Approved, Offers Hope for Children Living with Duchenne Muscular Dystrophy

By MDA Staff | October 26, 2023
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Clinical Trial Alert: Phase 2/3 Study of an Investigational Medication in Pediatric Patients with Generalized Myasthenia Gravis

By Sujatha Gurunathan | October 4, 2023
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Clinical Trial Alert: Phase 3 Study of Batoclimab in Adults with MG

By Sujatha Gurunathan | September 29, 2023
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New Therapy Brings Hope for Late-Onset Pompe Disease

By Melissa Donahue | September 21, 2023
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Deconstructing SMA Types and Treatments

By Sujatha Gurunathan | August 23, 2023
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FDA Approves Sarepta’s ELEVIDYS for Treatment of DMD

By Sujatha Gurunathan | June 22, 2023
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Gene Therapy Options Grow With New Drug Approvals

By Larry Luxner | May 18, 2023