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

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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 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 89 Issue: 17
  • Publication Date: 2017
  • Doi Number: 10.1212/wnl.0000000000004570
  • Journal Name: NEUROLOGY
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Page Numbers: pp.1811-1820
  • Hacettepe University Affiliated: Yes

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).