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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
Published on: December 20, 2017
Amy Anderson1, Jasvinder A Singh
1Medicine, Minneapolis VA Medical Center and University of Minnesota, One Veterans Drive, Minneapolis, MN, USA, 55417.
Pegloticase shows varied efficacy in reducing uric acid levels in chronic gout patients. However, no placebo-controlled trials exist, highlighting the need for more research on its safety and benefits.
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