نتایج جستجو برای: fabry disease

تعداد نتایج: 1493456  

2002
Yeong-Hau H. Lien

Fabry disease is a rare genetic disease due to a deficiency of the lysosomal hydrolase αgalactosidase A (α-Gal A) [1]. The recent advance of knowledge in Fabry disease can be applied to other lysosomal diseases and enhance our understanding in the pathogenesis and management of those once considered miserable and untreatable diseases. Unlike other lysosomal diseases, Fabry disease involves mult...

Journal: :Neurologia 2014
G Rodríguez-Boto M Rivero-Garvía R Gutiérrez-González A Pérez-Zamarrón J Vaquero

the formation of vascular microcalcifications. Presence of the sign has been correlated to age and severity of kidney disease, but not to the appearance of ischaemic stroke.5 The case we present confirms that T1-weighted images of bilateral pulvinar hyperintensity secondary to calcification are not pathognomonic for Fabry disease. Researchers require further studies to evaluate the frequency of...

2015
L. Choi J. Vernon O. Kopach M.S. Minett K. Mills P.T. Clayton T. Meert J.N. Wood

Fabry disease is an X-linked lysosomal storage disorder characterised by accumulation of glycosphingolipids, and accompanied by clinical manifestations, such as cardiac disorders, renal failure, pain and peripheral neuropathy. Globotriaosylsphingosine (lyso-Gb3), a deacylated form of globotriaosylceramide (Gb3), has emerged as a marker of Fabry disease. We investigated the link between Gb3, lys...

Journal: :BMC Cardiovascular Disorders 2002
David F Moore Gheona Altarescu Randall Pursley Umberto Campia Julio A Panza Emilios Dimitriadis Raphael Schiffmann

BACKGROUND Fabry disease is an X-linked recessive lysosomal storage disease resulting in the cellular accumulation of globotriaosylceramide particularly globotriaosylceramide. The disease is characterized by a dilated vasculopathy with arterial ectasia in muscular arteries and arterioles. Previous venous plethysomographic studies suggest enhanced endothelium-dependent vasodilation in Fabry dise...

2016
B Petritsch H Köstler A M Weng M Horn T Gassenmaier A S Kunz F Weidemann C Wanner T A Bley M Beer

BACKGROUND Fabry disease is characterized by a progressive deposition of sphingolipids in different organ systems, whereby cardiac involvement leads to death. We hypothesize that lysosomal storage of sphingolipids in the heart as occurring in Fabry disease does not reflect in higher cardiac lipid concentrations detectable by 1H magnetic resonance spectroscopy (MRS) at 3 Tesla. METHODS Myocard...

Journal: :Stroke 2010
Raf Brouns Vincent Thijs François Eyskens Marleen Van den Broeck Shibeshih Belachew Christine Van Broeckhoven Patricia Redondo Dimitri Hemelsoet Arnaud Fumal Sandrine Jeangette Werner Verslegers Robert Baker Derralynn Hughes Peter Paul De Deyn

BACKGROUND AND PURPOSE Data on the prevalence of Fabry disease in patients with central nervous system pathology are limited and controversial. In this study, we assessed the prevalence of Fabry disease in young patients presenting with cerebrovascular disease in Belgium. METHODS In this national, prospective, multicenter study, we screened for Fabry disease in 1000 patients presenting with i...

2012
Satoshi ISHII

Fabry disease is an inherited lysosomal storage disorder caused by deficient α-galactosidase A activity. Many missense mutations in Fabry disease often cause misfolded gene products, which leads to their retention in the endoplasmic reticulum by the quality control system; they are then removed by endoplasmic reticulum-associated degradation. We discovered that a potent α-galactosidase A inhibi...

2017
J. Politei C. Durand A.B. Schenone A. Torres J. Mukdsi B.L. Thurberg

Chronic intestinal pseudo-obstruction results in clinical manifestations that resemble intestinal obstruction but in the absence of any physical obstructive process. Fabry disease is an X-linked lysosomal storage disease characterized by the dysfunction of multiple systems, including significant gastrointestinal involvement. We report the occurrence of chronic intestinal pseudo-obstruction in t...

Journal: :Nephrology, dialysis, transplantation : official publication of the European Dialysis and Transplant Association - European Renal Association 2010
Agnes B Fogo Leif Bostad Einar Svarstad William J Cook Solange Moll Federic Barbey Laurette Geldenhuys Michael West Dusan Ferluga Bojan Vujkovac Alexander J Howie Aine Burns Roy Reeve Stephen Waldek Laure-Hélène Noël Jean-Pierre Grünfeld Carmen Valbuena João Paulo Oliveira Justus Müller Frank Breunig Xiao Zhang David G Warnock

BACKGROUND In Fabry nephropathy, alpha-galactosidase deficiency leads to accumulation of glycosphingolipids in all kidney cell types, proteinuria and progressive loss of kidney function. METHODS An international working group of nephrologists from 11 Fabry centres identified adult Fabry patients, and pathologists scored histologic changes on renal biopsies. A standardized scoring system was d...

2014
J. A. Matías-Guiu M. Yus M. Jorquera J. Porta-Etessam

the formation of vascular microcalcifications. Presence of the sign has been correlated to age and severity of kidney disease, but not to the appearance of ischaemic stroke. The case we present confirms that T1-weighted images of bilateral pulvinar hyperintensity secondary to calcification are not pathognomonic for Fabry disease. Researchers require further studies to evaluate the frequency of ...

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