Publication | Open Access

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Exon SkippingUrologyDuchenne Muscular DystrophyPhase 2MedicineOrthopaedic SurgeryNeuromusculoskeletal Disorder