title: Forty-Five Years of Duchenne Muscular Dystrophy in The Netherlands.
abstract: BACKGROUND
  Duchenne muscular dystrophy (DMD) is a progressive muscle disease. No curative therapy is currently available, but in recent decades standards of care have improved. These improvements include the use of corticosteroids and mechanical ventilation.
  
  
  OBJECTIVE
  To present a detailed population based report of the DMD disease course in The Netherlands (1980-2006) and evaluate the effect of changes in care by comparing it with an historical Dutch DMD cohort (1961-1974).
  
  
  METHODS
  Information about DMD patients was gathered through the Dutch Dystrophinopathy Database using a standardized questionnaire and information from treating physicians.
  
  
  RESULTS
  The study population involved 336 DMD patients (70% of the estimated prevalence), of whom 285 were still alive. Mean age at disease milestones was: diagnosis 4.3 years, wheelchair dependence 9.7 years, scoliosis surgery 14 years, cardiomyopathy (fractional shortening &lt;27%) 15 years, mechanical ventilation 17 years and death 19 years. Within our cohort, corticosteroid use was associated with an increased age of wheelchair dependence from 9.8 to 11.6 years (p &lt; 0.001). When comparing the recent cohort to the historical cohort, mean survival improved from 17 to 27 years (p &lt; 0.001).
  
  
  CONCLUSIONS
  The current study gives detailed information about the disease course of DMD patients, provides evidence for the positive effect of steroid treatment and mechanical ventilation and supports the use of patient registries as a valuable resource for evaluating improvements in care.
authors.affiliation: Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: J C
authors.initials: JC
authors.lastname: van den Bergen
authors.affiliation: Department of Clinical Genetics, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: H B
authors.initials: HB
authors.lastname: Ginjaar
authors.affiliation: Department of Genetics, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
authors.forename: A J
authors.initials: AJ
authors.lastname: van Essen
authors.affiliation: Rijndam Rehabilitation Center, Rotterdam, The Netherlands.
authors.forename: R
authors.initials: R
authors.lastname: Pangalila
authors.affiliation: Department of Rehabilitation, Radboud University Medical Center, Nijmegen, The Netherlands.
authors.forename: I J M
authors.initials: IJ
authors.lastname: de Groot
authors.affiliation: Department of Pulmonology/Center for Home Mechanical Ventilation, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
authors.forename: P J
authors.initials: PJ
authors.lastname: Wijkstra
authors.affiliation: Department of Intensive Care/Center for Home Mechanical Ventilation, University Medical Center, Erasmus Medical Center, Rotterdam, The Netherlands.
authors.forename: M P
authors.initials: MP
authors.lastname: Zijnen
authors.affiliation: Department of Pulmonology/Center for Home Mechanical Ventilation, Maastricht University Medical Center, Maastricht, The Netherlands.
authors.forename: N A M
authors.initials: NA
authors.lastname: Cobben
authors.affiliation: Center for Home Mechanical Ventilation, University Medical Center Utrecht, Utrecht, The Netherlands.
authors.forename: M J
authors.initials: MJ
authors.lastname: Kampelmacher
authors.affiliation: Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: B H A
authors.initials: BH
authors.lastname: Wokke
authors.affiliation: Department of Neurology, Erasmus University Medical Center, Rotterdam, The Netherlands.
authors.forename: I F M
authors.initials: IF
authors.lastname: de Coo
authors.affiliation: Department of Child Neurology, University of Groningen, University Medical Center Groningen, Groningen, The Netherlands.
authors.forename: J M
authors.initials: JM
authors.lastname: Fock
authors.affiliation: Dutch Association for Neuromuscular Diseases, Baarn, The Netherlands.
authors.forename: A M C
authors.initials: AM
authors.lastname: Horemans
authors.affiliation: Department of Rehabilitation, University Medical Center Utrecht, Utrecht, The Netherlands.
authors.forename: M
authors.initials: M
authors.lastname: van Tol
authors.affiliation: Duchenne Parent Project, The Netherlands.
authors.forename: E
authors.initials: E
authors.lastname: Vroom
authors.affiliation: Department of Paediatric Cardiology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: M E B
authors.initials: ME
authors.lastname: Rijlaarsdam
authors.affiliation: Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: C S M
authors.initials: CS
authors.lastname: Straathof
authors.affiliation: Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: E H
authors.initials: EH
authors.lastname: Niks
authors.affiliation: Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands.
authors.forename: J J G M
authors.initials: JJ
authors.lastname: Verschuuren