نتایج جستجو برای: gaucher type 1

تعداد نتایج: 3648308  

2017
Elma Aflaki Daniel K. Borger Richard J. Grey Martha Kirby Stacie Anderson Grisel Lopez Ellen Sidransky

Gaucher disease, the inherited deficiency of lysosomal glucocerebrosidase, is characterized by the presence of glucosylceramide-laden macrophages resulting from impaired digestion of aged erythrocytes or apoptotic leukocytes. Studies of macrophages from patients with type 1 Gaucher disease with genotypes N370S/N370S, N370S/L444P or N370S/c.84dupG revealed that Gaucher macrophages have impaired ...

2006
Young Bin Hong Eun Young Kim Sung-Chul Jung

Gaucher disease is caused by a deficiency of glucocerebrosidase. Patients with Gaucher disease are divided into three major phenotypes: chronic nonneuronopathic, acute neuronopathic, and chronic neuronopathic, based on symptoms of the nervous system, the severity of symptoms, and the age of disease onset. The characteristics of patients with acute neuronopathic- and chronic neuronopathic-type G...

Journal: :Journal of Medical Genetics 1988

2017
Jérôme Stirnemann Nadia Belmatoug Fabrice Camou Christine Serratrice Roseline Froissart Catherine Caillaud Thierry Levade Leonardo Astudillo Jacques Serratrice Anaïs Brassier Christian Rose Thierry Billette de Villemeur Marc G. Berger

Gaucher disease (GD, ORPHA355) is a rare, autosomal recessive genetic disorder. It is caused by a deficiency of the lysosomal enzyme, glucocerebrosidase, which leads to an accumulation of its substrate, glucosylceramide, in macrophages. In the general population, its incidence is approximately 1/40,000 to 1/60,000 births, rising to 1/800 in Ashkenazi Jews. The main cause of the cytopenia, splen...

Journal: :The American Journal of the Medical Sciences 1905

Journal: :Journal of the American Medical Association 1919

Journal: :Polskie Archiwum Medycyny Wewnetrznej 2014
Maciej Machaczka Alicja Markuszewska-Kuczyńska Sofie Regenthal Artur Jurczyszyn Krystyna Gałązka Björn E Wahlin Monika Klimkowska

INTRODUCTION In the absence of a known affected family member, frequent symptoms of Gaucher disease (GD), a rare lysosomal storage disorder, such as thrombocytopenia or splenomegaly, often lead to hematological diagnostic workup. OBJECTIVES The aim of the study was to compare the clinical utility of aspiration biopsy of the bone marrow (ASP) with trephine biopsy (TB) for the diagnosis of GD t...

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