Edward M. Kaye

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

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

Marco A. Passini, Jie Bu, Amy M. Richards et al. · Science Translational Medicine · 2011 · 548 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

Natural history of infantile‐onset spinal muscular atrophy

Stephen J. Kolb, Christopher S. Coffey, Jon Yankey et al. · Annals of Neurology · 2017 · 392 citations · Full text

Clinical research for rare disease: Opportunities, challenges, and solutions

Robert C. Griggs, Mark L. Batshaw, Mary Dunkle et al. · Molecular Genetics and Metabolism · 2008 · 390 citations · Full text

Clinical Population, Clinical Studies, Clinical Trials +3