Pivotal trial with plant cell-expressed recombinant glucocerebrosidase, taliglucerase alfa, a novel enzyme replacement therapy for Gaucher disease

Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience

(2010) A. Zimran et al.   BLOOD

Improving the accuracy of MRI spleen and liver volume measurements: A phase III Gaucher disease clinical trial setting as a model

(2010) Luc Bracoud et al.   BLOOD CELLS MOLECULES AND DISEASES

Focal splenic lesions in type I Gaucher disease are associated with poor platelet and splenic response to macrophage-targeted enzyme replacement therapy

(2010) Philip Stein et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Recommendations for treating patients with Gaucher disease with emerging enzyme products

(2009) Timothy M. Cox   BLOOD CELLS MOLECULES AND DISEASES

Force Majeure: Therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease

(2009) Carla E.M. Hollak et al.   BLOOD CELLS MOLECULES AND DISEASES

Dosing enzyme replacement therapy for Gaucher disease: older, but are we wiser?

(2009) Ellen Sidransky et al.   GENETICS IN MEDICINE

Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1

(2009) Gregory A Grabowski et al.   GENETICS IN MEDICINE

Drug Shortages and Public Health

(2009) Robert Steinbrook   NEW ENGLAND JOURNAL OF MEDICINE

A Plant-Derived Recombinant Human Glucocerebrosidase Enzyme—A Preclinical and Phase I Investigation

(2009) David Aviezer et al.   PLoS One

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

(2008) Neal Weinreb et al.   AMERICAN JOURNAL OF HEMATOLOGY

Phenotypic heterogeneity of N370S homozygotes with type I Gaucher disease: An analysis of 798 patients from the ICGG Gaucher Registry

(2008) C. Fairley et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Is the Drought Over for Pharming?

(2008) J. Kaiser   SCIENCE