Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up-Reference-Cited by-同舟云学术

Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up

Published:2016-05-10 Issue:4 Volume:54 Page:681-689
ISSN:0148-639X
Container-title:Muscle & Nerve
language:en
Short-container-title:Muscle Nerve

Author:

Connolly Anne M.¹²,Florence Julaine M.¹,Zaidman Craig M.¹²,Golumbek Paul T.¹²,Mendell Jerry R.³,Flanigan Kevin M.³,Karachunski Peter I.⁴,Day John W.⁵,McDonald Craig M.⁶,Darras Basil T.⁷,Kang Peter B.⁸,Siener Catherine A.¹,Gadeken Rebecca K.¹,Anand Pallavi¹,Schierbecker Jeanine R.¹,Malkus Elizabeth C.¹,Lowes Linda P.³,Alfano Lindsay N.³,Johnson Linda⁶,Nicorici Alina⁶,Kelecic Jason M.⁴,Quigley Janet⁷,Pasternak Amy E.⁷,Miller J. Philip⁹,

Affiliation:

1. Department of Neurology; Washington University School of Medicine; Saint Louis Missouri USA

2. Department of Pediatrics; Washington University School of Medicine; Saint Louis Missouri USA

3. Department of Pediatrics; Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital; Columbus Ohio USA

4. Department of Neurology; University of Minnesota; Minneapolis Minnesota USA

5. Department of Neurology; Stanford University; Stanford California USA

6. Department Physical Medicine and Rehabilitation; University of California, Davis Medical Center; Sacramento California USA

7. Department of Neurology; Harvard University, Boston Children's Hospital; Boston Massachusetts USA

8. Division of Pediatric Neurology; University of Florida College of Medicine; Gainesville Florida USA

9. Division of Biostatistics; Washington University School of Medicine; Saint Louis Missouri USA

Funder

MDA-DMD center

National Center for Research Resources

Paul D. Wellstone Muscular Dystrophy Clinical Research Center

Publisher

Wiley

Subject

Physiology (medical),Cellular and Molecular Neuroscience,Neurology (clinical),Physiology

Reference39 articles.

1. Dystrophin: the protein product of the Duchenne muscular dystrophy locus;Hoffman;Cell,1987

2. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy;Mendell;N Engl J Med,1989

3. Myostatin does not regulate cardiac hypertrophy or fibrosis;Cohn;Neuromuscul Disord,2007

4. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation;Eagle;Neuromuscul Disord,2002

5. Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy;Connolly;Muscle Nerve,2015

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