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- W2022337710 abstract "Fabry disease is a X-linked lysosomal storage disorder. Two preparations of the enzyme alpha-galactosidase A are available in Europe since 2001: agalsidase alpha and agalsidase beta. Clinical evidence of efficacy are mandatory considering the absence of a robust biomarker. A literature review was performed to assess the clinical efficacy of these two enzyme replacement therapies. Only open or randomised controlled trials were considered. No unflawed direct comparison exists between the two drugs. Significant clinical benefits have been demonstrated with enzyme replacement therapy (ERT), mainly at an early phase of the disease, with positive effects on heart, kidneys, pain, and quality of life. Further prospective studies are required to confirm the long term clinical benefits of ERT. More specific studies are also needed in women or with ERT earlier in the course of Fabry disease to assess prevention of organ damage." @default.
- W2022337710 created "2016-06-24" @default.
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- W2022337710 date "2009-10-01" @default.
- W2022337710 modified "2023-10-01" @default.
- W2022337710 title "Efficacité clinique de l’enzymothérapie dans la maladie de Fabry. Analyse critique" @default.
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- W2022337710 doi "https://doi.org/10.1016/j.revmed.2009.04.003" @default.
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