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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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| 出版年: | Mol Genet Metab Rep |
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| 主要な著者: | , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
Elsevier
2016
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5053031/ https://ncbi.nlm.nih.gov/pubmed/27747161 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2016.09.008 |
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