welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
recruiting

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

purpose:

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: December 10, 2019

rareRelated