Characteristics of 26 patients with type 3 Gaucher disease: A descriptive analysis from the Gaucher Outcome Survey

Characteristics of 26 patients with type 3 Gaucher disease: A descriptive analysis from the Gaucher Outcome Survey

The Gaucher Outcome Survey (GOS) is an international disease-specific registry established in 2010 for patients with a confirmed diagnosis of Gaucher disease (GD), regardless of GD type or treatment status. Historically, there has been a limited understanding of type 3 GD (GD3) and its natural histo...

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Bibliographic Details
Main Authors: Ida Vanessa D. Schwartz, Özlem Göker-Alpan, Priya S. Kishnani, Ari Zimran, Lydie Renault, Zoya Panahloo, Patrick Deegan
Format: Article
Language:English
Published: Elsevier 2018-03-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Type 3 Gaucher disease
Gaucher Outcome Survey
Velaglucerase alfa
Enzyme replacement therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426917301416

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http://www.sciencedirect.com/science/article/pii/S2214426917301416

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