C.M.M. is the study's principal investigator. E.K.H., R.T.A., and A.C. are study chairs.
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
Article first published online: 15 MAY 2013
Copyright © 2013 Wiley Periodicals, Inc.
Muscle & Nerve
Volume 48, Issue 1, pages 32–54, July 2013
How to Cite
McDonald, C. M., Henricson, E. K., Abresch, R. T., Han, J. J., Escolar, D. M., Florence, J. M., Duong, T., Arrieta, A., Clemens, P. R., Hoffman, E. P., Cnaan, A. and the Cinrg Investigators (2013), 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. Muscle Nerve, 48: 32–54. doi: 10.1002/mus.23807
This project was funded through grants from the U.S. Department of Education/NIDRR (H133B031118 and H133B090001), U.S. Department of Defense (W81XWH-09-1-0592), the National Institutes of Health (UL1RR031988, U54HD053177, UL1RR024992, U54RR026139, G12RR003051, 1R01AR061875, and RO1AR062380), and Parent Project Muscular Dystrophy.
Disclosures: The authors take full responsibility for the contents of this article, which do not represent the views of the U.S. Department of Education, the National Institutes of Health, the Department of Veterans Affairs, or the U.S. Government. R.T.A. has served as a consultant for PTC Therapeutics, Inc. A.A. has nothing to disclose. P.R.C. is a consultant for ReveraGen Biopharma. A.C. serves as a consultant for GlaxoSmithKline. T.D. has nothing to disclose. D.M.E. serves on the speakers bureau for and has received funding for travel and speaker honoraria from Athena Diagnostics, Inc., and also serves as a consultant for Acceleron Pharma, HALO Therapeutics, AVI Biopharma, the Gerson Lehman Group, and Medacorp. J.M.F. serves on a scientific advisory board for Prosensa, serves on the editorial board of Neuromuscular Disorders, and serves/has served as a member of the CINRG Executive Committee and as a consultant for Prosensa, GlaxoSmithKline, Genzyme Corporation, PTC Therapeutics, Inc., and Acceleron Pharma. E.K.H. is a member of the CINRG Executive Committee and has served as a consultant for Genzyme Corporation and PTC Therapeutics, Inc. J.J.H. has nothing to disclose. E.P.H. has served on advisory committees for AVI BioPharma, Inc., and as a consultant with Gerson Lehman Group, Medacorp, and Lazard Capital, and is a cofounder, board member, and shareholder of ReveraGen Biopharma. C.M.M has served on advisory committees for PTC Therapeutics, Inc., Sarepta Therapeutics, Inc., GlaxoSmithKline, plc, Prosensa, Halo Therapeutics, Shire HGT, and Novartis AG.
- Issue published online: 21 JUN 2013
- Article first published online: 15 MAY 2013
- Accepted manuscript online: 6 FEB 2013 10:15AM EST
- Manuscript Accepted: 30 JAN 2013
- follow-up study;
- health status;
- muscle strength/physiology;
- muscular dystrophies/classification;
- muscular dystrophies/Duchenne/physiopathology;
- muscular dystrophies/therapy;
- quality of life/psychology;
- respiratory function test
Contemporary natural history data in Duchenne muscular dystrophy (DMD) is needed to assess care recommendations and aid in planning future trials.
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.
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).
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. Muscle Nerve, 2013