Alglucosidase alfa treatment alleviates liver disease in a mouse model of glycogen storage disease type IV.

Published online

Journal Article

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.

Full Text

Duke Authors

Cited Authors

  • Yi, H; Gao, F; Austin, S; Kishnani, PS; Sun, B

Published Date

  • December 2016

Published In

Volume / Issue

  • 9 /

Start / End Page

  • 31 - 33

PubMed ID

  • 27747161

Pubmed Central ID

  • 27747161

International Standard Serial Number (ISSN)

  • 2214-4269

Digital Object Identifier (DOI)

  • 10.1016/j.ymgmr.2016.09.008


  • eng

Conference Location

  • United States