相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。The road to biosimilars in rare diseases - ongoing lessons from Gaucher disease
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BLOOD CELLS MOLECULES AND DISEASES (2018)
Causes of death in 184 patients with type 1 Gaucher disease from the United States who were never treated with enzyme replacement therapy
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BLOOD CELLS MOLECULES AND DISEASES (2018)
Hepatocellular carcinoma in Gaucher disease: an international case series
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JOURNAL OF INHERITED METABOLIC DISEASE (2018)
Prevalence and predictors of liver fibrosis evaluated by vibration controlled transient elastography in type 1 Gaucher disease
Fabio Nascimbeni et al.
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Transformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry
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AMERICAN JOURNAL OF HEMATOLOGY (2017)
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
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The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease
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Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3)
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ORPHANET JOURNAL OF RARE DISEASES (2015)
Gaucher Disease and Bone Manifestations
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CALCIFIED TISSUE INTERNATIONAL (2014)
The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) Statement: Guidelines for reporting observational studies
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INTERNATIONAL JOURNAL OF SURGERY (2014)
被撤回的出版物: World Medical Association Declaration of Helsinki Ethical Principles for Medical Research Involving Human Subjects (Retracted article. See vol. 135, pg. 2149, 2021)
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JOURNAL OF INHERITED METABOLIC DISEASE (2013)
Effects of imiglucerase on the growth and metabolism of Gaucher disease type I patients: a systematic review
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Characteristics of type I Gaucher disease associated with persistent thrombocytopenia after treatment with imiglucerase for 4-5 years
Carla E. M. Hollak et al.
BRITISH JOURNAL OF HAEMATOLOGY (2012)
Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply
Ari Zimran et al.
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JAK2V617F mutation and myeloproliferative malignancy in a patient with Type 1 Gaucher disease
Bryn D. Webb et al.
BLOOD CELLS MOLECULES AND DISEASES (2011)
Enzyme replacement therapy drug holiday: Results from an unexpected shortage of an orphan drug supply in Australia
Jack Goldblatt et al.
BLOOD CELLS MOLECULES AND DISEASES (2011)
Osseous Manifestations of Adult Gaucher Disease in the Era of Enzyme Replacement Therapy
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BRITISH JOURNAL OF HAEMATOLOGY (2009)
Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1
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GENETICS IN MEDICINE (2009)
A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase
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AMERICAN JOURNAL OF HEMATOLOGY (2008)
Overweight, insulin resistance and type II diabetes in type I Gaucher disease patients in relation to enzyme replacement therapy
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BLOOD CELLS MOLECULES AND DISEASES (2008)
Management of non-neuronopathic Gaucher disease with special reference to pregnancy, splenectomy, bisphosphonate therapy, use of biomarkers and bone disease monitoring
T. M. Cox et al.
JOURNAL OF INHERITED METABOLIC DISEASE (2008)
'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
M. Biegstraaten et al.
JOURNAL OF INHERITED METABOLIC DISEASE (2008)
Eight-Year Clinical Outcomes of Long-Term Enzyme Replacement Therapy for 884 Children With Gaucher Disease Type 1
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PEDIATRICS (2008)
The long-term international safety experience of imiglucerase therapy for Gaucher disease
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MOLECULAR GENETICS AND METABOLISM (2007)
Recommendations for the management of the haematological and onco-haematological aspects of Gaucher disease
Derralynn Hughes et al.
BRITISH JOURNAL OF HAEMATOLOGY (2007)
Quality of life related to type 1 Gaucher disease:: Spanish experience
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QUALITY OF LIFE RESEARCH (2005)
Therapeutic goals in the treatment of Gaucher disease
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SEMINARS IN HEMATOLOGY (2004)
Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: A report from the Gaucher Registry
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AMERICAN JOURNAL OF MEDICINE (2002)
The Gaucher registry -: Demographics and disease characteristics of 1698 patients with Gaucher disease
J Charrow et al.
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