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

Conclusions: Tadalafil did not lessen the decline in ambulatory ability in boys with DMD. Further studies should be considered to confirm the hypothesis-generating upper limb data and to determine whether ambulatory decline can be slowed by initiation of tadalafil before 7 years of age. Clinicaltrials.gov identifier: NCT01865084. Classification of evidence: This study provides Class I evidence that tadalafil does not slow ambulatory decline in 7- to 14-year-old boys with Duchenne muscular dystrophy.

http://www.neurology.org/cgi/content/short/89/17/1811?rss=1

Source: Neurology - October 23, 2017 Category: Neurology Authors: Victor, R. G., Sweeney, H. L., Finkel, R., McDonald, C. M., Byrne, B., Eagle, M., Goemans, N., Vandenborne, K., Dubrovsky, A. L., Topaloglu, H., Miceli, M. C., Furlong, P., Landry, J., Elashoff, R., Cox, D., For the Tadalafil DMD Study Group Tags: Muscle disease, Clinical trials Randomized controlled (CONSORT agreement) ARTICLE Source Type: research