A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys with Duchenne Muscular Dystrophy (DMD)

This study is an extension of the Phase II Dose Finding study evaluating the exon skipping intervention, NS-065/NCNP-01, targeted for the skipping of exon 53. The study will evaluate the tolerability and the effect of low and high IV doses of NS-065/NCNP-01 after an additional 24-week of weekly infusions. Patients who completed the NS-065/NCCNP-01 trial are eligible to enroll. To learn more about the study eligibility and participating sites please visit https://clinicaltrials.gov/ct2/show/NCT03167255 or contacts us at info@trinds.com.