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Therapeutic approaches in glycogen storage disease type II/pompe disease

Glycogen storage disease type II (GSDII)/Pompe disease is an autosomal recessive multi-system disorder due to a deficiency of the glycogen-degrading lysosomal enzyme, acid alpha-glucosidase. Without adequate levels of alpha-glucosidase, there is a progressive accumulation of glycogen inside the lyso...

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書目詳細資料
Main Authors: Schoser, Benedikt, Hill, Victoria, Raben, Nina
格式: Artigo
語言:Inglês
出版: Springer New York 2008
主題:
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2761605/
https://ncbi.nlm.nih.gov/pubmed/19019308
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nurt.2008.08.009
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