Motor and respiratory heterogeneity in Duchenne patients: implication for clinical trials.

doi:10.1016/j.ejpn.2011.07.001

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Article

Motor and respiratory heterogeneity in Duchenne patients: implication for clinical trials.

European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society - 1 Mar 2012

Humbertclaude Véronique, Hamroun Dalil, Bezzou Kamel, Bérard Carole, Boespflug-Tanguy Odile, Bommelaer Christine, Campana-Salort Emmanuelle, Cances Claude, Chabrol Brigitte, Commare Marie-Christine, Cuisset Jean-Marie, de Lattre Capucine, Desnuelle Claude, Echenne Bernard, Halbert Cécile, Jonquet Olivier, Labarre-Vila Annick, N'Guyen-Morel Marie-Ange, Pages Michel, Pepin Jean-Louis, Petitjean Thierry, Pouget Jean, Ollagnon-Roman Elisabeth, Richelme Christian, Rivier François, Sacconi Sabrina, Tiffreau Vincent, Vuillerot Carole, Picot Marie-Christine, Claustres Mireille, Béroud Christophe, Tuffery-Giraud Sylvie

Abstract excerpt

AIMS: Our objective was to clarify the clinical heterogeneity in Duchenne muscular dystrophy (DMD). METHODS: The French dystrophinopathy database provided clinical, histochemical and molecular data of 278 DMD patients (mean longitudinal follow-up: 14.2 years). Diagnosis was based on mutation identification in the DMD gene. Three groups were defined according to the age at ambulation loss: before 8 years (group...

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PMID: 21920787
DOI: 10.1016/j.ejpn.2011.07.001

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