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.
authors.affiliation: Department of Physical Medicine & Rehabilitation, School of Medicine, University of California, Davis, 4860 Y Street, Suite 3850, Sacramento, California 95817, USA. cmmcdonald@ucdavis.edu
authors.forename: Craig M
authors.initials: CM
authors.lastname: McDonald
authors.forename: Erik K
authors.initials: EK
authors.lastname: Henricson
authors.forename: R Ted
authors.initials: RT
authors.lastname: Abresch
authors.forename: Jay J
authors.initials: JJ
authors.lastname: Han
authors.forename: Diana M
authors.initials: DM
authors.lastname: Escolar
authors.forename: Julaine M
authors.initials: JM
authors.lastname: Florence
authors.forename: Tina
authors.initials: T
authors.lastname: Duong
authors.forename: Adrienne
authors.initials: A
authors.lastname: Arrieta
authors.forename: Paula R
authors.initials: PR
authors.lastname: Clemens
authors.forename: Eric P
authors.initials: EP
authors.lastname: Hoffman
authors.forename: Avital
authors.initials: A
authors.lastname: Cnaan
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005938:Glucocorticoids / Q000627:therapeutic use*; D006801:Humans; D007391:International Cooperation; D038622:Internationality; D008137:Longitudinal Studies; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000453:epidemiology*; D011446:Prospective Studies; D012107:Research Design; D055815:Young Adult
chemical_list: D005938:Glucocorticoids