Although exon skipping strategies have been applied almost exclusively for dystrophin mutations, they might also be applicable for other genetic disorders where protein truncation is compatible ...

during the process of RNA splicing, causing the body to produce more dystrophin. One of the few other therapeutics focused on exon 53 skipping is NS Pharma’s Viltepso. At present, the market for ...

Elevidys was granted expanded approval by the U.S. Food and Drug Administration in 2024, with expectations high that the therapy, designed to restore a critical muscle protein called dystrophin ...