Article: article from journal or magazin.
Essais thérapeutiques dans la dystrophie musculaire de Duchenne: entre espoirs et désespoirs [Therapeutic trials for Duchenne muscular dystrophy: between hopes and disappointments].
Revue Médicale Suisse
Publication types: English Abstract ; Journal Article Publication Status: ppublish
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.
Child, Gene Therapy, Humans, Muscular Dystrophy, Duchenne/drug therapy, Muscular Dystrophy, Duchenne/genetics
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