FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook
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Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disorder caused by dystrophin deficiency. Antisense oligonucleotide (ASO)-mediated exon skipping has emerged as a cornerstone of DMD therapy to restore dystrophin expression. This review provides ... ...
