Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV.
Mol Genet Metab Rep
; 9: 31-33, 2016 Dec.
Article
in En
| MEDLINE
| ID: mdl-27747161
ABSTRACT
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 of rhGAA at 40 mg/kg for 4 weeks significantly reduced hepatic glycogen accumulation, lowered liver/body weight ratio, and reduced plasma ALP and ALT activities in GSD IV mice. Our data suggests that rhGAA is a potential therapy for GSD IV.
ALT, alanine aminotransferase; AST, aspartate aminotransferase; Alglucosidase alfa; ERT, enzyme replacement therapy; GAA, acid α-glucosidase; GBE, glycogen branching enzyme; GSD IV, glycogen storage disease type IV; Glycogen storage disease type IV; Liver; M6PR, mannose-6-phosphate receptor; Recombinant human acid-α glucosidase
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Language:
En
Journal:
Mol Genet Metab Rep
Year:
2016
Document type:
Article
Affiliation country:
United States
Publication country:
EEUU
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ESTADOS UNIDOS
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ESTADOS UNIDOS DA AMERICA
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EUA
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UNITED STATES
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UNITED STATES OF AMERICA
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US
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USA