title: [Therapeutic trials for Duchenne muscular dystrophy: between hopes and disappointments].
abstract: Duchenne muscular dystrophy is an X-linked progressive muscle disease. Since the discovery of the dystrophin gene responsible for the condition, various therapeutic strategies have been elaborated. In this paper we introduce three of them, which are well into clinical trials. The first is based on the ability to read through premature stop codons, the second is based on the technique of exon skipping. Both strategies are examples of "personalized medicines", tailored for specific mutation types. The third approach is a pharmacological one, potentially useful for all Duchenne patients, regardless of their mutation type. These first clinical trials raise many questions for researchers as well as for patients and their families, some of which are discussed.
authors.affiliation: Consultation des maladies neuromusculaires, Unité de neuropédiatrie et de neuroréhabilitation pédiatrique, DMCP, CHUV, 1011 Lausanne.
authors.forename: Clemens
authors.initials: C
authors.lastname: Bloetzer
authors.forename: Joël
authors.initials: J
authors.lastname: Fluss
authors.forename: Pierre-Yves
authors.initials: PY
authors.lastname: Jeannet
mesh_terms: D002648:Child; D015316:Genetic Therapy; D006801:Humans; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000235:genetics