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Written by: Pr Brigitte Chabrol and Pr Isabelle Desguerre

Disclosure of interest: B. Chabrol: Clinical trials: as main (head) clinical or laboratory investigator, or study coordinator (PTC124) and Clinical trials: as co-investigator or study contributor (BioMarin) ; Expert reports (Board PTC, Board Biomarin, Board Shire); Conferences: attendance as audience member (BioMarin, Genzyme, Shire). I. Desguerre: fees for congress, training program, expert board (PTC Inc, GSK, Servier); Clinical trials: as main (head) clinical or laboratory investigator, or study coordinator (PTC Inc and GSK).

Publication of this content is the sole responsibility of the publisher and the editorial board of the journal. This document is distributed by PCT Therapeutics as a service with the aim of informing health professionals. 
The sponsor was not involved in writing the document.

Medical Coordinator: Dr Catherine Romain.
Marketing Manager: Benoît Sibaud b.sibaud@elsevier.com.
Project Manager: Marie Sadoux.
Design: Jennifer Brial
Photo credits: Cover and backgrounds.

©Elsevier Masson SAS. 2015. This document is intended for use solely by health professionals.

 

For more information:

  • Chelly J, Desguerre I, Progressive muscular dystrophies (Chapter 141). Handb Clin Neurol 2013;113:1343-66.
  • Desguerre I, Christov C, Mayer M, et al. Clinical heterogeneity of Duchenne muscular dystrophy (DMD): definition of sub-phenotypes and predictive criteria by long-term follow-up. PLoS One 2009;4:e4347.
  • Humbertclaude V, Hamroun D, Bezzou K, et al. Motor and respiratory heterogeneity in Duchenne patients: implication for clinical trials. Eur J Paediatr Neurol 2012;16:149-60.
  • Sabouraud P, Cuisset JM, Cances C, et al.; Commission Maladies Neuromusculaires [Neuromuscular Disease Board] of the Société Française de Neurologie Pédiatrique [French Paediatric Neurology Society]. Diagnostic approach of hyperCKemia in childhood. Arch Pediatr 2009;16:678-80.
  • Bushby K et al. The Diagnosis and Management of Duchenne Muscular Dystrophy. Part 1: Lancet Neurol 2010; 9:77-93. Part 2: Lancet Neurol 2010; 9:177-89.
  • Muscular dystrophies: From Duchenne to Becker. Arch Pediatr 2015;22(12 Suppl 1):12S1-72..