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- W2518191778 endingPage "36" @default.
- W2518191778 startingPage "30" @default.
- W2518191778 abstract "Anderson-Fabry disease is a potentially life-threatening hereditary lysosomal storage disorder taking origin in over 1,000 known pathogenic mutations in the alpha-galactosidase A encoding gene. Over the past 15 years, intravenous replacement therapy of the deficient alpha agalsidase A enzyme has been well-established retarding the progression of a multisystemic disease and organ involvement. Despite this innovative treatment approach, premature deaths still do occur. The response to enzyme replacement therapy (ERT) varies considerably and appears to depend on gender, genotype (classic or later onset/non-classic), stage of disease or age and agalsidase inhibition by anti-agalsidase antibodies. Early ERT treatment at young age, a personalized approach, and adjunctive therapies for specific disease manifestations appear to impact on prognosis and are currently favored with the expectance of more effective intravenous and oral treatments in the short future." @default.
- W2518191778 created "2016-09-16" @default.
- W2518191778 creator A5012012015 @default.
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- W2518191778 date "2016-01-01" @default.
- W2518191778 modified "2023-10-16" @default.
- W2518191778 title "Long Term Treatment with Enzyme Replacement Therapy in Patients with Fabry Disease" @default.
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- W2518191778 doi "https://doi.org/10.1159/000448968" @default.
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- W2518191778 hasPublicationYear "2016" @default.
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