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- W2024995046 abstract "Récemment, des progrès considérables ont été réalisés dans le domaine des maladies lysosomales. Dans le passé aucun traitement spécifique n’était disponible pour les patients ; la prise en charge consistait en un traitement symptomatique et au traitement des complications. Progressivement des traitements spécifiques de substitution enzymatique qualifiés de « médicaments orphelins » ont fait leur apparition pour certaines de ces maladies et plus particulièrement pour la maladie de Gaucher, la maladie de Fabry, les mucopolysaccharidoses de type I, II et VI et pour la maladie de Pompe. Dans cette revue nous allons résumer les bénéfices cliniques et les inconvénients de chacune de ces thérapeutiques et nous allons également décrire de nouvelles perspectives thérapeutiques en cours de développement dans le domaine de l’enzymothérapie substitutive. In the last years, much progress has been achieved in the treatment of lysosomal storage disorders. Until recently only symptomatic treatment was available for the affected patients. Progressively enzyme replacement treatments have been developed for several diseases, namely Gaucher disease, Fabry disease, mucopolysaccharidoses type I, II and VI and Pompe disease. In this review we will summarize the efficacy and safety of these treatments and describe new therapeutic trials for other lysosomal storage disorders or perspectives in the use of currently available treatments." @default.
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- W2024995046 date "2011-10-01" @default.
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- W2024995046 title "Le traitement par enzymothérapie des maladies lysosomales" @default.
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- W2024995046 doi "https://doi.org/10.1016/j.arcped.2011.07.002" @default.
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