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Tagged: exon skipping therapy

Drug Development for Rare Duchenne Muscular Dystrophy (DMD)

April 20th, 2021 | exon skipping therapy

Progress and limitation of therapeutic approaches targeting DMD By: Dr. Maria Niu Duchenne muscular dystrophy (DMD) is an X—a chromosome-linked recessive disorder caused by mutations in the gene coding muscle cytoskeletal protein dystrophin. Because of the X-linked inheritance, nearly 1 in 3,500 males are affected worldwide, while females occasionally develop symptoms. There is no curative […]

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