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

2015497 citationsJournal Articlebronze Open Access

Authors

Jerry R. Mendell · Ohio State University Hospital

Nathalie Goemans

Linda Lowes · Nationwide Children's Hospital

Lindsay N. Alfano · Muscular Dystrophy Association

Katherine Berry · Nationwide Children's Hospital

James Shao · Sarepta Therapeutics (United States)

Edward M. Kaye · Sarepta Therapeutics (United States)

Abstract

Over 3 years of follow-up, eteplirsen-treated patients showed a slower rate of decline in ambulation assessed by 6MWT compared to untreated matched HC.

Topics & Keywords

Muscle Physiology and Disorders Exercise and Physiological Responses Muscle metabolism and nutrition

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Annals of Neurology

Volume 79, Issue 2, pp. 257-271

DOI: 10.1002/ana.24555

Field-Weighted Citation Impact: 23.80