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Large animal models and new therapies for glycogen storage disease

โœ Scribed by Brooks, Elizabeth D.; Koeberl, Dwight D.

Book ID
127389672
Publisher
Springer
Year
2014
Tongue
English
Weight
194 KB
Volume
38
Category
Article
ISSN
0141-8955

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## Abstract There are more than 40 different forms of inherited lysosomal storage diseases (LSDs) known to occur in humans and the aggregate incidence has been estimated to approach 1 in 7000 live births. Most LSDs are associated with high morbidity and mortality and represent a significant burden