@article{37b38be477e248d5801a094055e8b1fb,
title = "Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA–DMD network follow-up",
abstract = "Introduction: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Methods: Subjects (n = 91; 16.8 ± 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score ≤4, using repeated measures. Results: Eight patients (12–33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here. Muscle Nerve 54: 681–689, 2016.",
keywords = "Brooke Scale, Duchenne muscular dystrophy, Egen Klassifikation Scale, clinical trial, force vital capacity, non-ambulatory, outcomes",
author = "{the MDA–DMD Clinical Research Network} and Connolly, {Anne M.} and Florence, {Julaine M.} and Zaidman, {Craig M.} and Golumbek, {Paul T.} and Mendell, {Jerry R.} and Flanigan, {Kevin M.} and Karachunski, {Peter I.} and Day, {John W.} and McDonald, {Craig M.} and Darras, {Basil T.} and Kang, {Peter B.} and Siener, {Catherine A.} and Gadeken, {Rebecca K.} and Pallavi Anand and Schierbecker, {Jeanine R.} and Malkus, {Elizabeth C.} and Lowes, {Linda P.} and Alfano, {Lindsay N.} and Linda Johnson and Alina Nicorici and Kelecic, {Jason M.} and Janet Quigley and Pasternak, {Amy E.} and Miller, {J. Philip}",
note = "Funding Information: The authors acknowledge the other members of the MDA–DMD network who contributed to this project: Charlie O. Wulf, BA (Department of Neurology, Washington University School of Medicine, Saint Louis, Missouri); Erica Goude, BA (Department Physical Medicine and Rehabilitation, University of California, Davis Medical Center, Sacramento, California); Joline C. Dalton, MS, CGC (Department of Neurology, University of Minnesota, Minneapolis, Minnesota); Laurence Viollet-Callendret, PhD (Department of Pediatrics, Ohio State University, and the Center for Gene Therapy, Nationwide Children's Hospital, Columbus, Ohio); Karen K. Buser, MS (Department of Neurology, University of Minnesota, Minneapolis, Minnesota); Elizabeth Shriber, BA (Department of Neurology, Harvard University, Boston Children's Hospital, Boston Massachusetts); and Rebecca Parad, BA (Department of Neurology, Harvard University, Boston Children's Hospital, Boston, Massachusetts). Publisher Copyright: {\textcopyright} 2016 Wiley Periodicals, Inc.",
year = "2016",
month = oct,
day = "1",
doi = "10.1002/mus.25089",
language = "English",
volume = "54",
pages = "681--689",
journal = "Muscle and Nerve",
issn = "0148-639X",
number = "4",
}