Substrate reduction therapy

Substrate reduction therapy

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The glycosphingolipidoses are a family of storage diseases that arise due to incomplete catabolism of glycosphingolipids (GSLs) in the lysosome (Wraith, 2002). The majority are autosomal recessive disorders and result from mutations in the genes that encode the catabolic enzymes of the lysosome (Win...

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Dettagli Bibliografici
Autori principali:	Platt, F, Butters, T
Natura:	Book section
Pubblicazione:	Springer US 2007

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