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; Anne M. Connolly

doi:10.1164/rccm.201703-0507WS

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
, Anne M. Connolly

Department of Neurology

Research output: Contribution to journal › Article › peer-review

44 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 language	English
Pages (from-to)	512-519
Number of pages	8
Journal	American journal of respiratory and critical care medicine
Volume	196
Issue number	4
DOIs	https://doi.org/10.1164/rccm.201703-0507WS
State	Published - Aug 15 2017

Keywords

Duchenne muscular dystrophy
Outcome
Respiratory failure

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10.1164/rccm.201703-0507WS

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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., 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

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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",

author = "Jonathan Finder and Mayer, \{Oscar Henry\} and Daniel Sheehan and Hemant Sawnani and Abresch, \{R. Ted\} and Joshua Benditt and Birnkrant, \{David J.\} and Tina Duong and Erik Henricson and Kathi Kinnett and McDonald, \{Craig M.\} and Connolly, \{Anne M.\}",

note = "Publisher Copyright: Copyright {\textcopyright} 2017 by the American Thoracic Society.",

year = "2017",

month = aug,

day = "15",

doi = "10.1164/rccm.201703-0507WS",

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AU - Finder, Jonathan

AU - Mayer, Oscar Henry

AU - Sheehan, Daniel

AU - Sawnani, Hemant

AU - Abresch, R. Ted

AU - Benditt, Joshua

AU - Birnkrant, David J.

AU - Duong, Tina

AU - Henricson, Erik

AU - Kinnett, Kathi

AU - McDonald, Craig M.

AU - Connolly, Anne M.

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AB - 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.

KW - Duchenne muscular dystrophy

KW - Outcome

KW - Respiratory failure

UR - https://www.scopus.com/pages/publications/85028608968

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EP - 519

JO - American journal of respiratory and critical care medicine

JF - American journal of respiratory and critical care medicine

IS - 4

ER -

Pulmonary endpoints in duchenne muscular dystrophy a workshop summary

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Keywords

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