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Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options

Background: Gaucher disease is a rare lysosomal storage disease resulting from a deficiency or reduced activity in the acid β-glucocosidase enzyme. Only 1 treatment option was available for 15 years, but several new treatment options have come to market since 2003. Objective: The article will detail...

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Detalhes bibliográficos
Publicado no:Hosp Pharm
Main Authors: Van Rossum, Alison, Holsopple, Megan
Formato: Artigo
Idioma:Inglês
Publicado em: Thomas Land Publishers, Inc. 2016
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4981103/
https://ncbi.nlm.nih.gov/pubmed/27559188
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1310/hpj5107-553
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