Article
Clinical heterogeneity of duchenne muscular dystrophy (DMD): definition of sub-phenotypes and predictive criteria by long-term follow-up.
PloS one - 1 Jan 2009
Desguerre Isabelle, Christov Christo, Mayer Michele, Zeller Reinhard, Becane Henri-Marc, Bastuji-Garin Sylvie, Leturcq France, Chiron Catherine, Chelly Jamel, Gherardi Romain K
Abstract excerpt
BACKGROUND: To explore clinical heterogeneity of Duchenne muscular dystrophy (DMD), viewed as a major obstacle to the interpretation of therapeutic trials METHODOLOGY/PRINCIPAL FINDINGS: A retrospective single institution long-term follow-up study was carried out in DMD patients with both complete lack of muscle dystrophin and genotyping. An exploratory series (series 1) was used to assess phenotypic...
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