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

201835 citationsJournal Articlegold Open Access

Authors

Craig M. McDonald · University of California, Davis

Brenda Wong · Cincinnati Children's Hospital Medical Center

Kevin M. Flanigan · Nationwide Children's Hospital

Rosamund Wilson

S. de Kimpe · BioMarin (Netherlands)

Afrodite Lourbakos · BioMarin (Netherlands)

Zhengning Lin · BioMarin (Netherlands)

Abstract

Drisapersen 6 mg/kg/week for 24 weeks resulted in a treatment benefit in 6MWD, largely maintained 24 weeks off-treatment. This study provided insights for further studies to optimize dosage regimen.

Topics & Keywords

Muscle Physiology and Disorders Exercise and Physiological Responses Coenzyme Q10 studies and effects

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Annals of Clinical and Translational Neurology

Volume 5, Issue 8, pp. 913-926

DOI: 10.1002/acn3.579

Field-Weighted Citation Impact: 1.95