Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical trials

Author:

Elstein Deborah,Mellgard Björn,Dinh Quinn,Lan Lan,Qiu Yongchang,Cozma Claudia,Eichler Sabrina,Böttcher Tobias,Zimran Ari

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference36 articles.

1. Gaucher disease and related lysosomal storage diseases;Zimran,2016

2. Clinical manifestations and management of Gaucher disease;Linari;Clin. Cases Miner. Bone Metab.,2015

3. Biomarkers;Lorne,2013

4. Hyperferritinemia and iron overload in type 1 Gaucher disease;Stein;Am. J. Hematol.,2010

5. Improvement of bone disease by imiglucerase (Cerezyme) therapy in patients with skeletal manifestations of type 1 Gaucher disease: results of a 48-month longitudinal cohort study;Sims;Clin. Genet.,2008

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