Browsing Duke Scholarly Works by Subject "M6PR, mannose-6-phosphate receptor"
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Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV. (Mol Genet Metab Rep, 2016-12)Patients with progressive hepatic form of GSD IV often die of liver failure in early childhood. We tested the feasibility of using recombinant human acid-α glucosidase (rhGAA) for treating GSD IV. Weekly intravenously injection ...