Giles Campion

A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

Nathalie Goemans, Eugenio Mercuri, Hirofumi Komaki et al. · Neuromuscular Disorders · 2017 · 128 citations · Full text

Medicine, Molecular Medicine, Neuromuscular Disorders +7

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

Nathalie Goemans, M. Tulinius, M. van den Hauwe et al. · PLoS ONE · 2016 · 89 citations · Full text

Medicine, Pharmacokinetics, Pharmacology +13

Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy

Eugenio Mercuri, James Signorovitch, Elyse Swallow et al. · Neuromuscular Disorders · 2016 · 72 citations · Full text

Medicine, Pediatrics, Rehabilitation Medicine +15

Placebo‐controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy

Craig M. McDonald, Brenda Wong, Kevin M. Flanigan et al. · Annals of Clinical and Translational Neurology · 2018 · 36 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +13

Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids

Nathalie Goemans, M. Tulinius, Anna‐Karin Kroksmark et al. · Neuromuscular Disorders · 2016 · 19 citations · Full text

Medicine, Clinical Sciences, Neuromuscular Disorders +8