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Hematopoietic stem cell transplantation for Gaucher disease.

著者 Somaraju UR , Tadepalli K
Cochrane Database Syst Rev.2017 Oct 18 ; 10():CD006974.
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Gaucher disease is the most common lysosomal storage disorder caused by a deficiency of the enzyme glucocerebrosidase. Current treatment of the disease involves a choice from enzyme replacement therapy, substrate reduction therapy and hemotopoietic stem cell transplantation (HSCT). HSCT is a high risk procedure with possible long-term benefits in the regression of skeletal and neurological changes in people with Gaucher disease. This is an update of a previously published Cochrane Review.
PMID: 29044482 [PubMed - as supplied by publisher]
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