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- W170091617 abstract "We describe the pathway to the development of enzyme replacement therapy (ERT) for Fabry disease with particular emphasis on the agalsidase alfa preparation. For both agalsidase alfa and beta enzyme preparations, initial studies were performed in a mouse model of Fabry disease. Subsequent placebo-controlled and open-label trials showed reduction in neuropathic pain and gastrointestinal symptoms and increased cold perception and sweating in patients with the disorder. Partial reversal of the vascular pathophysiology was also observed. Reports from uncontrolled studies suggest a cardiac benefit as well, and initial studies in women with Fabry disease suggest a decreased disease burden in those patients who are receiving ERT. Most encouraging are the data on safety and initial symptomatic efficacy of ERT in children as young as 7 years of age. We conclude that ERT in Fabry disease has a definite benefit. Long-term studies are necessary to assess to what extent it delays the occurrence of sentinel disease manifestations, such as cardiac events, end-stage renal disease, stroke and death." @default.
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- W170091617 date "2006-01-01" @default.
- W170091617 modified "2023-09-26" @default.
- W170091617 title "Development of enzyme replacement therapy for Fabry disease" @default.
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