Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.

Eric P HoffmanBenjamin D SchwartzLaurel J Mengle-GawEdward C SmithDiana CastroJean K MahCraig M McDonaldNancy L KuntzRichard S FinkelMichela GuglieriKatharine BushbyMar TuliniusYoram NevoMonique M RyanRichard WebsterAndrea L SmithLauren P MorgenrothAdrienne ArrietaMaya ShimonyCatherine SienerMark JarosPhil ShaleJohn M McCallKanneboyina NagarajuJohn van den AnkerLaurie S ConklinAvital CnaanHeather Gordish-DressmanJesse M DamskerPaula R Clemensnull null

Published in: Neurology (2019)

This study provides Class IV evidence that for boys with DMD, vamorolone demonstrated possible efficacy compared to a natural history cohort of glucocorticoid-naive patients and appeared to be tolerated.

Keyphrases

duchenne muscular dystrophy
end stage renal disease
ejection fraction
chronic kidney disease
newly diagnosed
clinical trial
prognostic factors
muscular dystrophy
randomized controlled trial
hiv infected
phase iii
patient reported outcomes