Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

2011660 citationsJournal Articlebronze Open Access

Authors

Nathalie Goemans · KU Leuven

M. Tulinius · University of Gothenburg

Johanna T. van den Akker

Brigitte E. Burm · ProQR Therapeutics (Netherlands)

Peter F. Ekhart · ProQR Therapeutics (Netherlands)

Niki Heuvelmans · ProQR Therapeutics (Netherlands)

Tjadine M. Holling · ProQR Therapeutics (Netherlands)

Abstract

Systemically administered PRO051 showed dose-dependent molecular efficacy in patients with Duchenne's muscular dystrophy, with a modest improvement in the 6-minute walk test after 12 weeks of extended treatment. (Funded by Prosensa Therapeutics; Netherlands National Trial Register number, NTR1241.).

Topics & Keywords

Muscle Physiology and Disorders Viral Infections and Immunology Research Telomeres, Telomerase, and Senescence

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: New England Journal of Medicine

Volume 364, Issue 16, pp. 1513-1522

DOI: 10.1056/nejmoa1011367

Field-Weighted Citation Impact: 42.59