Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment

A Cross-Sectional Retrospective Study of Non-Splenectomized and Never-Treated Patients with Type 1 Gaucher Disease

(2020) Christine Serratrice et al.   Journal of Clinical Medicine

Liver steatosis is highly prevalent and is associated with metabolic risk factors and liver fibrosis in adult patients with type 1 Gaucher disease

(2020) Fabio Nascimbeni et al.   LIVER INTERNATIONAL

Gaucher Disease in Bone: From Pathophysiology to Practice

(2019) Derralynn Hughes et al.   JOURNAL OF BONE AND MINERAL RESEARCH

The road to biosimilars in rare diseases ‐ ongoing lessons from Gaucher disease

(2019) Guillermo Drelichman et al.   AMERICAN JOURNAL OF HEMATOLOGY

Gaucher Disease and Myelofibrosis: A Combined Disease or a Misdiagnosis?

(2018) Sabrina Mariani et al.   ACTA HAEMATOLOGICA

Growth and final height of children with Gaucher disease: A 15-year follow-up at an Israeli Gaucher center

(2018) Espen Mendelsohn et al.   BLOOD CELLS MOLECULES AND DISEASES

Causes of death in 184 patients with type 1 Gaucher disease from the United States who were never treated with enzyme replacement therapy

(2018) Neal J Weinreb et al.   BLOOD CELLS MOLECULES AND DISEASES

Hepatocellular carcinoma in Gaucher disease: an international case series

(2018) Martine Regenboog et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Prevalence and predictors of liver fibrosis evaluated by vibration controlled transient elastography in type 1 Gaucher disease

(2018) Fabio Nascimbeni et al.   MOLECULAR GENETICS AND METABOLISM

Spleen Size Is Significantly Influenced by Body Height and Sex: Establishment of Normal Values for Spleen Size at US with a Cohort of 1200 Healthy Individuals

(2016) Kai Uwe Chow et al.   RADIOLOGY

The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease

(2015) Carla E.M. Hollak et al.   BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM

Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3)

(2015) Neal J. Weinreb et al.   Orphanet Journal of Rare Diseases

Gaucher Disease and Bone Manifestations

(2014) Gemma Marcucci et al.   CALCIFIED TISSUE INTERNATIONAL

The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) Statement: Guidelines for reporting observational studies

(2014) Erik von Elm et al.   International Journal of Surgery

World Medical Association Declaration of Helsinki

(2013)   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Effects of imiglucerase on the growth and metabolism of Gaucher disease type I patients: a systematic review

(2013) Divair Doneda et al.   Nutrition & Metabolism

Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years

(2012) Carla E. M. Hollak et al.   BRITISH JOURNAL OF HAEMATOLOGY

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

(2012) Neal J. Weinreb et al.   JOURNAL OF INHERITED METABOLIC DISEASE

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

(2011) Patrick B. Deegan et al.   MEDICINE

Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply

(2010) Ari Zimran et al.   BLOOD CELLS MOLECULES AND DISEASES

JAK2V617F mutation and myeloproliferative malignancy in a patient with Type 1 Gaucher disease

(2010) Bryn D. Webb et al.   BLOOD CELLS MOLECULES AND DISEASES

Enzyme replacement therapy “drug holiday”: Results from an unexpected shortage of an orphan drug supply in Australia

(2010) Jack Goldblatt et al.   BLOOD CELLS MOLECULES AND DISEASES

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

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

Eight-Year Clinical Outcomes of Long-Term Enzyme Replacement Therapy for 884 Children With Gaucher Disease Type 1

(2008) H. Andersson et al.   PEDIATRICS

Overweight, insulin resistance and type II diabetes in type I Gaucher disease patients in relation to enzyme replacement therapy

(2007) M. Langeveld et al.   BLOOD CELLS MOLECULES AND DISEASES