Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary

Jonathan Finder, Oscar Henry Mayer, Daniel Sheehan, Hemant Sawnani, R. Ted Abresch, Joshua Benditt, David J. Birnkrant, Tina Duong, Erik K Henricson, Kathi Kinnett, Craig M McDonald, Anne M. Connolly

Research output: Contribution to journalReview article

23 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)512-519
Number of pages8
JournalAmerican Journal of Respiratory and Critical Care Medicine
Volume196
Issue number4
DOIs
StatePublished - Aug 15 2017

Keywords

  • Duchenne muscular dystrophy
  • outcome
  • respiratory failure

ASJC Scopus subject areas

  • Medicine(all)
  • Pulmonary and Respiratory Medicine
  • Critical Care and Intensive Care Medicine

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    Finder, J., Mayer, O. H., Sheehan, D., Sawnani, H., Abresch, R. T., Benditt, J., Birnkrant, D. J., Duong, T., Henricson, E. K., Kinnett, K., McDonald, C. M., & Connolly, A. M. (2017). Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary. American Journal of Respiratory and Critical Care Medicine, 196(4), 512-519. https://doi.org/10.1164/rccm.201703-0507WS