• Am. J. Respir. Crit. Care Med. · Aug 2017

    Review

    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 Henricson, Kathi Kinnett, Craig M McDonald, and Anne M Connolly.
    • 1 Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania.
    • Am. J. Respir. Crit. Care Med. 2017 Aug 15; 196 (4): 512-519.

    AbstractDevelopment 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.

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