Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance

Pompe disease, which results from mutations in the gene encoding the glycogen-degrading lysosomal enzyme acid α-glucosidase (GAA) (also called “acid maltase”), causes death in early childhood related to glycogen accumulation in striated muscle and an accompanying infantile-onset cardiomyopathy. The...

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Auteurs principaux: Sun, Baodong , Bird, Andrew , Young, Sarah P. , Kishnani, Priya S. , Chen, Y.-T. , Koeberl, Dwight D. 
Format: Artigo
Langue:Inglês
Publié: American Society of Human Genetics 2007
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Article
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC2265658/
https://ncbi.nlm.nih.gov/pubmed/17924344
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