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RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying Strategies

2026-04-28

Abstract excerpt

RNA-based therapeutics are reshaping the treatment landscape for skeletal muscle disorders by enabling modulation of RNA processing or direct correction of disease-causing alleles. In Duchenne muscular dystrophy (DMD), four antisense oligonucleotides—eteplirsen, golodirsen, viltolarsen, and casimersen—have received FDA approval; these phosphorodiamidate morpholino oligomers (PMOs) induce exon skipping to restore t...

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Literature Corpus work
1b777e92-e00e-59f4-824a-50e404b72844
DOI
10.20944/preprints202604.1918.v1
Open publication

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RNA Therapeutics Targeting Skeletal Muscle: Emerging Antisense and Gene-Modifying StrategiesDOI 10.20944/preprints202604.1918.v1
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