Lindsay N. Alfano

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.2K citations · Full text

Eteplirsen for the treatment of Duchenne muscular dystrophy

Jerry R. Mendell, Louise R. Rodino‐Klapac, Zarife Sahenk et al. · Annals of Neurology · 2013 · 770 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

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

Jerry R. Mendell, Zarife Sahenk, Kelly J. Lehman et al. · JAMA Neurology · 2020 · 360 citations · Full text