A. A. Agafonova

Clinical experience of treating 28 patients with Duchenne muscular dystrophy with the exon 45 skipping drug casimersen

Background. Duchenne muscular dystrophy (DMD) is an irreversible, progressive fatal genetic disorder caused by the absence of functional dystrophin protein in muscle due to mutations in the DMD gene. Exon-skipping therapy for DMD aims to increase the amount of functionally active protein dystrophin. Casimersen, a drug...