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Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
study id #: NCT02667483
condition: Duchenne Muscular Dystrophy
status: active, not recruiting
purpose:This is a phase I/II study to evaluate the safety, tolerability, efficacy, and pharmacokinetic (PK) profile of DS-5141b in patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 skipping and to determine the dosage for subsequent studies.
intervention: DS-5141b
mechanism of action: Exon-skipping to promote dystrophin production
results: https://clinicaltrials.gov/ct2/show/results/NCT02667483
last updated: July 08, 2019
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