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Optimal therapy in Gaucher disease

Gaucher disease (GD), the inherited deficiency of the lysosomal enzyme glucocerebrosidase, presents with a wide range of symptoms of varying severity, and primarily affects the skeletal, hematologic and nervous systems. To date, the standard of care has included enzyme replacement therapy with imigl...

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Autor principal: Goker-Alpan, Ozlem
Format: Artigo
Idioma:Inglês
Publicat: Dove Medical Press 2010
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2909498/
https://ncbi.nlm.nih.gov/pubmed/20668714
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