Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
E Mercuri, JJ Vilchez, O Boespflug-Tanguy… - The Lancet …, 2024 - thelancet.com
Background Duchenne muscular dystrophy, the most common childhood muscular
dystrophy, is caused by dystrophin deficiency. Preclinical and phase 2 study data have …
dystrophy, is caused by dystrophin deficiency. Preclinical and phase 2 study data have …
269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy–What have we learned? 9–11 December 2022, Hoofddorp, The …
KJ Naarding, G Stimpson, SJ Ward, N Goemans… - Neuromuscular …, 2023 - Elsevier
There are multiple avenues for therapeutic development in Duchenne muscular dystrophy
(DMD), which are highlighted in the first section of this report for the “10 years of Clinical …
(DMD), which are highlighted in the first section of this report for the “10 years of Clinical …