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Exon skipping and dystrophin restoration in patientswith Duchenne muscular dystrophy after systemicphosphorodiamidate morpholino oligomer treatment:an open-label, phase 2, dose-escalation study
This phase 2, open-label trial evaluated AVI-4658 (PMO) therapy in 19 boys with Duchenne muscular dystrophy (DMD), assessing exon 51 skipping and dystrophin restoration. The drug was well tolerated, showing dose-dependent dystrophin expression, with significant improvements at higher doses. Increased dystrophin correlated with protein localization and reduced inflammation, though responses varied. Findings support AVI-4658 as a potential disease-modifying treatment, emphasizing the need for long-term trials to confirm clinical benefits.
