Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
Edward C SmithLaurie S ConklinEric P HoffmanPaula R ClemensJean K MahRichard S FinkelMichela GuglieriMar TuliniusYoram NevoMonique M RyanRichard WebsterDiana CastroNancy L KuntzLaurie KerchnerLauren P MorgenrothAdrienne ArrietaMaya ShimonyMark JarosPhil ShaleHeather Gordish-DressmanLaura HagertyUtkarsh J DangJesse M DamskerBenjamin D SchwartzLaurel J Mengle-GawCraig M McDonaldnull nullPublished in: PLoS medicine (2020)
Clinical trials were registered at www.clinicaltrials.gov, and the links to each trial are as follows (as provided in manuscript text): VBP15-002 [NCT02760264] VBP15-003 [NCT02760277] VBP15-LTE [NCT03038399].