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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Bibliografiske detaljer
Main Authors: Angelini, Corrado, Semplicini, Claudio, Tonin, Paola, Filosto, Massimiliano, Pegoraro, Elena, Sorarù, Gianni, Fanin, Marina
Format: Artigo
Sprog:Inglês
Udgivet: SAGE Publications 2009
Fag:
Original Research
Online adgang: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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