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Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1

Bouwien E Smid1, Maria J Ferraz2, Marri Verhoek3

  • 1Department of Endocrinology and Metabolism, Academic Medical Centre, Amsterdam, The Netherlands.

Orphanet Journal of Rare Diseases
|March 25, 2016
PubMed
Abstract

Keywords:
ChitotriosidaseEliglustatEnzyme replacement therapyGaucher diseaseGlucosylceramideGlucosylsphingosineMiglustat

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