Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary

Am J Respir Crit Care Med. 2017 Aug 15;196(4):512-519. doi: 10.1164/rccm.201703-0507WS.

Abstract

Development of novel therapeutics for treatment of Duchenne muscular dystrophy (DMD) has led to clinical trials that include pulmonary endpoints that allow assessment of respiratory muscle status, especially in nonambulatory subjects. Parent Project Muscular Dystrophy (PPMD) convened a workshop in Bethesda, Maryland, on April 14 and 15, 2016, to summarize published respiratory data in DMD and give guidance to clinical researchers assessing the effect of interventions on pulmonary outcomes in DMD.

Keywords: Duchenne muscular dystrophy; outcome; respiratory failure.

Publication types

  • Review

MeSH terms

  • Humans
  • Lung / physiopathology*
  • Muscular Dystrophy, Duchenne / physiopathology*
  • Respiratory Muscles / physiopathology*