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Study of SRP-4045 and SRP-4053 in DMD Patients

key information

study id #: NCT02500381

condition: Duchenne Muscular Dystrophy

status: recruiting


The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

intervention: SRP-4045, SRP-4053, Placebo

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT02500381

last updated: September 19, 2020