A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

Victor, Ronald; Sweeney, H.; Finkel, Richard; McDonald, Craig; Byrne, Barry; Eagle, Michelle; Goemans, Nathalie; Vandenborne, Krista; Dubrovsky, Alberto; Topaloglu, Haluk; Miceli, M.; Furlong, Pat; Landry, John; Elashoff, Robert; Cox, David

doi:10.1212/wnl.0000000000004570

A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

Victor R. G. , Sweeney H. L. , Finkel R., McDonald C. M. , Byrne B., Eagle M., ...More

NEUROLOGY, vol.89, no.17, pp.1811-1820, 2017 (Journal Indexed in SCI)

Publication Type: Article / Article
Volume: 89 Issue: 17
Publication Date: 2017
Doi Number: 10.1212/wnl.0000000000004570
Title of Journal : NEUROLOGY
Page Numbers: pp.1811-1820

Abstract

Objective: To conduct a randomized trial to test the primary hypothesis that once-daily tadalafil, administered orally for 48 weeks, lessens the decline in ambulatory ability in boys with Duchenne muscular dystrophy (DMD).