Clinical Trials

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Clinical Study Alert: Phase 2 Study of Brogidirsen in Boys with DMD

By Sujatha Gurunathan | March 29, 2024
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Clinical Study Alert: A Phase 1/2 Study of Safety and Efficacy of LX2006 Gene Therapy in Adults with Cardiomyopathy Associated with Friedreich Ataxia

By Sujatha Gurunathan | March 25, 2024
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FDA’s Approval of Duvyzat (givinostat) Offers New Hope to People Living with Duchenne Muscular Dystrophy

By MDA Staff | March 21, 2024
The key pathologic change of the muscular dystrophy is the myonecrosis. At an early phase, necrotic fibers appear swollen, homogeneous and deeply eosinophilic. This disease is caused by mutations of dystrophin, the largest known human gene, located on chromosome Xq21.
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Simply Stated: Updates in LAMA2 Muscular Dystrophy

By Sujatha Gurunathan | February 20, 2024
Illustration of a black metal pipeline on a yellow background
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What Is the Drug Development Pipeline?

By Amy Bernstein | February 12, 2024
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In Case You Missed It…

By MDA Staff | February 8, 2024
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9 Questions to Ask Before Joining a Clinical Trial

By Myrna Traylor | January 11, 2024
Color image of a real life young physically impaired ALS patient computer gaming with the help of his electronic wheelchair.
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Simply Stated: Updates in Collagen VI-Related Dystrophy

By Sujatha Gurunathan | January 6, 2024
Mitochondrion in cell cross section
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Simply Stated: Research Updates in Leigh Syndrome

By Sujatha Gurunathan | December 6, 2023
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Clinical Trial Alert: Phase 3 Study of Brepocitinib in Adults with Dermatomyositis

By Sujatha Gurunathan | November 16, 2023