نتایج جستجو برای: gaucher type 1

تعداد نتایج: 3648308  

2004
JACK GOLDBLATT

Type I Gaucher disease, the subject of this article, was initially reported by Gaucher' in 1882 as a non-leukaemic splenic epithelioma. The biochemical defect, an autosomal recessively inherited lysosomal glucocerebrosidase enzyme deficiency, was delineated in 1965,2 3 and more recently the full length coding DNA sequence has been cloned and characterised.4 Gaucher disease is conventionally cla...

Journal: :iranian journal of child neurology 0
hadi mozafari 1. department of clinical biochemistry, faculty of medical sciences, tarbiat modares university, tehran, iran mohammad taghikhani 1. department of clinical biochemistry, faculty of medical sciences, tarbiat modares university, tehran, iran shohreh khatami 2. department of biochemistry, pasteur institute of iran, tehran, iran mohammad reza alaei 3. department of pediatric, faculty of medicine, shahid beheshti university of medical sciences, tehran, iran asad vaisi-raygani 4. department of clinical biochemistry, medical school, kermanshah university of medical sciences, kermanshah, iran zohreh rahimi 4. department of clinical biochemistry, medical school, kermanshah university of medical sciences, kermanshah, iran

how to cite this article: mozafari h, taghikhani m, khatami sh, alaei mr, vaisi-raygani a, rahimi z. chitotriosidase activity and gene polymorphism in iranian patients with gaucher disease and sibling carriers. iran j child neurol. autumn 2016; 10(4):62-70. abstract objective chitotriosidase (ct) activity is a useful biomarker for diagnosis and monitoring of gaucher disease (gd). its applicatio...

Journal: :Philosophical Transactions of the Royal Society of London. Series B: Biological Sciences 2003

Journal: :Journal of Inherited Metabolic Disease 2010

Journal: :Brain : a journal of neurology 2010
Marieke Biegstraaten Eugen Mengel Laszlo Maródi Milan Petakov Claus Niederau Pilar Giraldo Derralyn Hughes Mirando Mrsic Atul Mehta Carla E M Hollak Ivo N van Schaik

Type 1 Gaucher disease is currently categorized as non-neuronopathic, although recent studies suggest peripheral neurological manifestations. We report prevalence and incidence data for peripheral neuropathy and associated conditions from a multinational, prospective, longitudinal, observational cohort study in patients with type 1 Gaucher disease, either untreated or receiving enzyme replaceme...

2018
Dominick Amato Mary Anne Patterson

BACKGROUND Intravenous enzyme replacement therapy is a first-line therapy for Gaucher disease type 1, and substrate reduction therapy represents an oral treatment alternative. Both enzyme replacement therapy and substrate reduction therapy are generally used as monotherapies in Gaucher disease. However, one randomized study and several case reports have described combination therapy over short ...

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