A Phase 2, Open-Label Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)

Duchenne Muscular Dystrophy

Incidence and severity of treatment emergent adverse events (TEAEs)., Changes in vital sign measurements., Changes in clinical laboratory results., Changes in electrocardiogram (ECG) parameters., Changes in physical examination findings.

Plasma concentration of study drug compounds and their final metabolite., Change from parent study Part A and OL Period baselines to LTE EOS in 10-Meter Walk/Run (10MWR)., Change from parent study Part A and OL Period baselines to LTE EOS in timed rise from floor (TRF)., Change from parent study Part A and OL Period baselines to LTE EOS in Timed 4-Stair Climb (4SC)., Change from parent study Part A and OL Period baselines to LTE EOS in stride velocity 95th centile (SV95C)., Change from parent study Part A and OL Period baselines to LTE EOS in North Star Ambulatory Assessment (NSAA)., Change from parent study Part A and OL Period baselines to LTE EOS in Performance of the Upper Limb v2.0 (PUL 2.0)., Anti-drug antibody (ADA) and anti-dystrophin antibody in serum.

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