Progress in Enzyme Replacement Therapy in Glycogen Storage Disease Type II

Glycogen storage disease type II (GSDII) is an autosomal recessive lysosomal disorder caused by mutations in the gene encoding alpha-glucosidase (GAA). The disease can be clinically classified into three types: a severe infantile form, a juvenile and an adultonset form. Cases with juvenile or adult...

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Main Authors: Angelini, Corrado, Semplicini, Claudio, Tonin, Paola, Filosto, Massimiliano, Pegoraro, Elena, Sorarù, Gianni, Fanin, Marina
Formáid: Artigo
Teanga:Inglês
Foilsithe: SAGE Publications 2009
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Original Research
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC3002626/
https://ncbi.nlm.nih.gov/pubmed/21179524
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1756285609103324
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