@article{43052609f3ad4bbab91da8c5dc4953a7,
title = "The Cooperative International Neuromuscular Research Group Duchenne Natural History Study-A Longitudinal Investigation In The Era Of Glucocorticoid Therapy: Design Of Protocol And The Methods Used",
abstract = "Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials. Methods: The Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DMD-NHS) enrolled 340 individuals, aged 2-28 years, with DMD in a longitudinal, observational study at 20 centers. Assessments obtained every 3 months for 1 year, at 18 months, and annually thereafter included: clinical history; anthropometrics; goniometry; manual muscle testing; quantitative muscle strength; timed function tests; pulmonary function; and patient-reported outcomes/health-related quality-of-life instruments. Results: Glucocorticoid (GC) use at baseline was 62% present, 14% past, and 24% GC-naive. In those ≥6 years of age, 16% lost ambulation over the first 12 months (mean age 10.8 years). Conclusions: Detailed information on the study methodology of the CINRG DMD-NHS lays the groundwork for future analyses of prospective longitudinal natural history data. These data will assist investigators in designing clinical trials of novel therapeutics.",
keywords = "Adolescent, Adult, Child/preschool, Follow-up study, Health status, Human, Locomotion, Male, Muscle strength/physiology, Muscular dystrophies/Duchenne/physiopathology, Muscular dystrophies/classification, Muscular dystrophies/therapy, Phenotype, Quality of life/psychology, Respiratory function test",
author = "{the CINRG Investigators} and McDonald, {Craig M.} and Henricson, {Erik K.} and Abresch, {R. Ted} and Han, {Jay J.} and Escolar, {Diana M.} and Florence, {Julaine M.} and Tina Duong and Adrienne Arrieta and Clemens, {Paula R.} and Hoffman, {Eric P.} and Avital Cnaan and V. Vishwanathan and S. Chidambaranathan and Biggar, {W. Douglas} and Mah, {Jean K.} and Mar Tulinius and Robert Leshner and Carolina Tesi-Rocha and Andrew Korn-berg and Monique Ryan and Yoram Nevo and Alberto Dubrovsky and Nancy Kuntz and Sherilyn Driscoll and Anne Connolly and Alan Pestronk and Jean Teasley and Tulio Bertorini and Kathryn North and Hanna Kolski and Jose Carlo and Ksenija Gorni and Timothy Lotze and John Day and Josh Benditt and Louis Boitano and David Birnkrant and David Connuck and Jonathan Finder and Veronica Hinton and Katherine Mathews and Richard Moxley and Susan Sparks and Erynn Gordon and Michelle Cregan and Erica Goude and Merete Glick and Linda Johnson and Nanette Joyce and Alina Nicorici",
year = "2013",
month = jul,
day = "1",
doi = "10.1002/mus.23807",
language = "אנגלית",
volume = "48",
pages = "32--54",
journal = "Muscle and Nerve",
issn = "0148-639X",
publisher = "John Wiley & Sons Inc.",
number = "1",
}