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Report Summary

Preclinical Development of New Therapy for Glycogen Storage Diseases


Study Summary

Overall Study Design 
 
This is an overview of Glycogen Storage Disease GSD GSD is caused by the inherited deficiency of an enzyme involved 
in either the degradation or synthesis of glycogen More than 10 such conditions have been characterized the features of 
the well characterized GSDs are discussed in this publication Experiments with gene therapy have demonstrated proof-of-
concept in several GSDs in animal models 
 

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    Lorraine Matheson
    NGVB Biorepository Manager
    317-274-4519
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