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Exon-Skipping in Duchenne Muscular Dystrophy
Exon-skipping therapy aims to restore dystrophin production in Duchenne muscular dystrophy (DMD) by using antisense oligonucleotides (AONs) to bypass mutations. Eteplirsen and viltolarsen target exon 51, producing a truncated but functional dystrophin protein. While promising, challenges remain, including low dystrophin levels and variable patient responses. Gene therapy and stop codon read-through drugs offer alternative strategies. Early intervention and combination therapies may improve outcomes for DMD patients.
