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active, not recruiting

Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

key information

study id #: NCT02667483

condition: Duchenne Muscular Dystrophy

status: active, not recruiting


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: June 24, 2020