Progress in enzyme replacement therapy in glycogen storage disease type II

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 adult-onset form. Cases with juvenile or adult...

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Bibliographic Details
Main Authors: Corrado Angelini, Claudio Semplicini, Paola Tonin, Massimiliano Filosto, Elena Pegoraro, Gianni Sorarù, Marina Fanin
Format: Article
Language:English
Published: SAGE Publishing 2009-05-01
Series:Therapeutic Advances in Neurological Disorders
Online Access:https://doi.org/10.1177/1756285609103324

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https://doi.org/10.1177/1756285609103324

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