نتایج جستجو برای: gaucher disease

تعداد نتایج: 1490299  

2013
Junitsu Ito Takafumi Saito Chikahiko Numakura Akiko Iwaba Shinpei Sugahara Rika Ishii Chikako Sato Hiroaki Haga Kazuo Okumoto Yuko Nishise Hisayoshi Watanabe Hiroyuki Ida Kiyoshi Hayasaka Hitoshi Togashi Sumio Kawata Yoshiyuki Ueno

A 21-year-old man with a history of sudden rectal hemorrhage was referred to our hospital. Examination disclosed thrombocytopenia and hepatosplenomegaly. A liver biopsy specimen demonstrated Gaucher cells in Glisson's capsule. Additional investigations revealed a low level of leukocyte β-glucosidase activity and common mutations of the glucocerebrosidase gene, L444P/D409H. We diagnosed the pati...

Journal: :Bulletin of the NYU hospital for joint diseases 2007
Kevin F Lutsky Nirmal C Tejwani

Gaucher disease is a rare, hereditary disease caused by lack of a lysosomal enzyme. This results in the accumulation of glucocerebroside in the cells of the reticuloendothelial system, including the bone marrow. The orthopaedic manifestations of this disease are important for the orthopaedic surgeon to recognize and understand. Patients with Gaucher disease are at risk for pathologic fracture, ...

Journal: :Journal of gynecology and womens health 2023

2016
Jennifer Ibrahim Lisa H. Underhill John S. Taylor Jennifer Angell M. Judith Peterschmitt

Eliglustat is a recently approved oral therapy in the United States and Europe for adults with Gaucher disease type 1 who are CYP2D6 extensive, intermediate, or poor metabolizers (> 90% of patients) that has been shown to decrease spleen and liver volume and increase hemoglobin concentrations and platelet counts in untreated adults with Gaucher disease type 1 and maintain these parameters in pa...

Journal: :Journal of medical genetics 1996
E Sidransky N Tayebi B K Stubblefield W Eliason A Klineburgess G P Pizzolato J N Cox J Porta A Bottani C D DeLozier-Blanchet

It has recently been emphasised that a subset of patients with type 2 Gaucher disease die in the neonatal period. This report describes an Afghani family with two conceptuses having severe, prenatally detected Gaucher disease. Mutational analysis showed that the family carried a known complex allele which included mutations at amino acids L444P, A456P, and V460V. Although glucocerebrosidase RNA...

Journal: :The Journal of bone and joint surgery. British volume 2008
G Kenet E Ezra S Wientroub D M Steinberg N Rosenberg D Waldman S Hayek

The role of heritable thrombophilic risk factors in the pathogenesis of the Perthes' disease is controversial. The clinical and radiological findings of Perthes' disease may be indistinguishable from those of Gaucher's disease, and the most common Jewish N370S Gaucher mutation is threefold greater in patients with Perthes' disease. Familial osteonecrosis of the femoral head is associated with v...

Journal: :Journal of Medical Genetics 1988

Journal: :Blood 1969
H E Kattlove J C Williams E Gaynor M Spivack R M Bradley R O Brady

G AUCHER’S DISEASE is an inherited disorder1 of sphingolipid nietabolism caused by a deficiency of glucocerebrosidase.2”-’ The deficiency of this enzyme leads to the accumulation of its substrate, glucocerebroside,3 in reticuloendothelial cells, imparting to them their characteristic appearance. The presence of Gaucher cells was considered pathognomonic of this hereditary disorder until 1966 at...

2015
Violeta Streanga Cristina Jitareanu Irina M. Ciomaga Doina Mihaila Nicolai Nistor

Gaucher disease is the most common lysosomal storage disorder, with autosomal recessive transmission. The disease is due to glucocerebrosidase enzyme defi ciency, resulting in accumulation of glucocerebroside in all organs. The diagnosis is established by measuring enzyme activity. Among the clinical forms, type 2 is the rarest and has the most dismal prognosis. We present the case of an infant...

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