Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment

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

(2011) A. Zimran et al.   BLOOD

Osseous Manifestations of Adult Gaucher Disease in the Era of Enzyme Replacement Therapy

(2011) Patrick B. Deegan et al.   MEDICINE

Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders

(2011) Carla EM Hollak et al.   Orphanet Journal of Rare Diseases

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

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

Vascular complications after splenectomy for hematologic disorders

(2009) S. E. Crary et al.   BLOOD

Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis

(2009) Pramod K. Mistry et al.   BRITISH JOURNAL OF HAEMATOLOGY

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

(2009) KB Sims et al.   CLINICAL GENETICS

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

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

Management of non-neuronopathic Gaucher disease with special reference to pregnancy, splenectomy, bisphosphonate therapy, use of biomarkers and bone disease monitoring

(2008) T. M. Cox et al.   JOURNAL OF INHERITED METABOLIC DISEASE

‘Non-neuronopathic’ Gaucher disease reconsidered. Prevalence of neurological manifestations in a Dutch cohort of type I Gaucher disease patients and a systematic review of the literature

(2008) M. Biegstraaten et al.   JOURNAL OF INHERITED METABOLIC DISEASE