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Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD

key information

study id #: NCT04060199

condition: Duchenne Muscular Dystrophy

status: recruiting


The main objective of this study is to evaluate the efficacy of Viltolarsen compared to placebo in Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping.

intervention: Viltolarsen, Placebo

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT04060199

last updated: October 31, 2020