This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.
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Part 1: Overall Summary of Treatment-emergent Adverse Events (TEAEs)
Timeframe: Baseline up to Week 24
Part 1: Area Under the Plasma Concentration Versus Time Curve (AUC) of NS-050/NCNP-03
Timeframe: Day 1 (1st dose) for each dose level
Part 1: Amount of Drug Excreted in Urine of NS-050/NCNP-03
Timeframe: Day 1 (1st dose) for each dose level
Part 2: Change from baseline in skeletal muscle dystrophin protein by immunoblot (Western blot)
Timeframe: Baseline, Week25