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

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...

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Detalles Bibliográficos
Autores principales: Yi, Haiqing, Gao, Fengqin, Austin, Stephanie, Kishnani, Priya S, Sun, Baodong
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2016
Materias:
Short Communication
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5053031/
https://www.ncbi.nlm.nih.gov/pubmed/27747161
http://dx.doi.org/10.1016/j.ymgmr.2016.09.008

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5053031/
https://www.ncbi.nlm.nih.gov/pubmed/27747161
http://dx.doi.org/10.1016/j.ymgmr.2016.09.008

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