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In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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α-Galactosidase A-deficient rats accumulate glycosphingolipids and develop cardiorenal phenotypes of Fabry disease

James J Miller1, Kazuhiro Aoki2, Carly A Mascari1

  • 1Department of Biochemistry, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.

FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology
|July 7, 2018
PubMed

Keywords:
animal modelglycobiologyheartkidneylysosomal storage disease

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