Nathalie Goemans

Local Dystrophin Restoration with Antisense Oligonucleotide PRO051

Judith C. van Deutekom, Anneke A. M. Janson, Ieke B. Ginjaar et al. · New England Journal of Medicine · 2007 · 760 citations

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

Nathalie Goemans, M. Tulinius, Johanna T. van den Akker et al. · New England Journal of Medicine · 2011 · 659 citations · Full text

Longitudinal effect of eteplirsen versus historical control on ambulation in <scp>D</scp> uchenne muscular dystrophy

Jerry R. Mendell, Nathalie Goemans, Linda Lowes et al. · Annals of Neurology · 2015 · 495 citations · Full text

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Craig M. McDonald, Craig Campbell, Ricardo Erazo Torricelli et al. · The Lancet · 2017 · 446 citations · Full text

Rare Diseases, Urology, Clinical Trials +5

Ataluren treatment of patients with nonsense mutation dystrophinopathy

K. Bushby, Richard S. Finkel, Brenda Wong et al. · Muscle & Nerve · 2014 · 416 citations · Full text