Browsing Biological Sciences by Author "Westwood, Stephanie"
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Evaluating enzyme replacement therapies for Anderson-Fabry disease : commentary on a recent report
Giugliani, Roberto; Westwood, Stephanie; Wellhoefer, Hartmann; Schenk, Jörn Magnus; Gurevich, Andrey; Kampmann, Christoph (2018) [Journal article]Anderson-Fabry disease (AFD) is a rare lysosomal storage disorder. Randomized controlled clinical trials (RCTs) are preferred as the highest category of evidence, but limited availability of robust evidence in rare diseases ...