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

Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary.

Author information

1
1 Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania.
2
2 Department of Pediatrics, University of Pennsylvania School of Medicine, Philadelphia, Pennsylvania.
3
3 Department of Pediatrics, University at Buffalo, The State University of New York, Buffalo, New York.
4
4 Division of Pulmonology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio.
5
5 Department of Physical Medicine and Rehabilitation and.
6
6 Division of Pulmonary and Critical Care Medicine, University of Washington School of Medicine, Seattle, Washington.
7
7 MetroHealth Medical Center, Cleveland, Ohio.
8
8 Case Western Reserve University, Cleveland, Ohio.
9
9 Department of Neurology, Stanford University, Stanford, California.
10
10 Parent Project Muscular Dystrophy, Hackensack, New Jersey; and.
11
11 Department of Pediatrics, University of California Davis, Sacramento, California.
12
12 Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri.

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

PMID:
28636407
DOI:
10.1164/rccm.201703-0507WS
[Indexed for MEDLINE]

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