نتایج جستجو برای: fabry

تعداد نتایج: 6280  

2012
MI HEE LEE EUN NAM CHOI YEO JIN JEON SUNG-CHUL JUNG

Fabry disease is a lysosomal storage disorder (LSD) caused by deficiency of α-galactosidase A (α-gal A), resulting in deposition of globotriaosylceramide (Gb3; also known as ceramide trihexoside) in the vascular endothelium of many organs. A gradual accumulation of Gb3 leads to cardiovascular, cerebrovascular and renal dysfunction. Endothelial cel...

2013
J. Oden P. Delaye N. Dubreuil

J. Oden Laboratoire Charles Fabry, Institut d’Optique, CNRS, Univ Paris Sud, 2 Avenue Augustin Fresnel, 91127 PALAISEAU cedex, FRANCE S. Trebaol Laboratoire Charles Fabry, Institut d’Optique, CNRS, Univ Paris Sud, 2 Avenue Augustin Fresnel, 91127 PALAISEAU cedex, FRANCE P. Delaye Laboratoire Charles Fabry, Institut d’Optique, CNRS, Univ Paris Sud, 2 Avenue Augustin Fresnel, 91127 PALAISEAU cede...

2017
Marco Spada David Kasper Veronica Pagliardini Elisa Biamino Silvana Giachero Francesco Porta

BACKGROUND Fabry disease is an X-linked lysosomal storage disorder due to α-galactosidase A (α-Gal A) deficiency. Clinical onset of Fabry disease is preceded by significant storage of globotriaosylceramide (Gb3) and related glycosphingolipids, but the extent of the metabolic progression before symptoms is unknown. Using a newly recognized effector and marker of Fabry disease, globotriaosylsphin...

Journal: :JIMD reports 2013
Markus Niemann Arndt Rolfs Anne Giese Hermann Mascher Frank Breunig Georg Ertl Christoph Wanner Frank Weidemann

The X-chromosomal-linked lysosomal storage disorder Fabry disease can lead to life-threatening manifestations. The pathological significance of the Fabry mutation D313Y is doubted, because, in general, D313Y patients do not present clinical manifestations conformable with Fabry disease. This is in contrast to the analysis of the alpha-galactosidase A activity, which is reduced in D313Y patients...

Journal: :European journal of echocardiography : the journal of the Working Group on Echocardiography of the European Society of Cardiology 2011
Gerald Mundigler Martina Gaggl Georg Heinze Senta Graf Manfred Zehetgruber Natalija Lajic Till Voigtländer Christine Mannhalter Raute Sunder-Plassmann Eduard Paschke Günter Fauler Gere Sunder-Plassmann

AIMS The binary sign, a binary appearance of the left ventricular endocardial border, was suggested to be an echocardiographic hallmark in diagnosing Fabry disease, a hereditary, lysosomal storage disorder. The aim of the present study was to examine the reliability of the binary sign as a screening tool to identify patients with Fabry disease. METHODS AND RESULTS In total 309 subjects with a...

2013
Sachie Nakano Yoshihito Morizane Noriko Makisaka Toshihiro Suzuki Tadayasu Togawa Takahiro Tsukimura Ikuo Kawashima Hitoshi Sakuraba Futoshi Shibasaki

Fabry disease is an X-linked genetic disorder caused by defects in the α-galactosidase A (GLA) gene, and heterogeneous mutations lead to quantitative and/or qualitative defects in GLA protein in male patients with Fabry disease. Random X-chromosomal inactivation modifies the clinical and biochemical features of female patients with Fabry disease. Functional polymorphisms have been frequently re...

Journal: :Noro psikiyatri arsivi 2015
Çetin Kürşad Akpinar Hande Türker Oytun Bayrak Nilgün Cengiz

INTRODUCTION Fabry disease (FD) is an X-linked recessive inherited disorder characterized by lysosomal alpha-galactosidase deficiency. The purpose of our study was to assess and compare the electroneuromyographic (ENMG) findings of 15 patients with Fabry disease and the electroneurographic (ENG) findings of 15 healthy controls. We have not encountered any similar study in the medical literature...

2017
Jin-Ho Choi Beom Hee Lee Sun Hee Heo Gu-Hwan Kim Yoo-Mi Kim Dae-Seong Kim Jung Min Ko Young Bae Sohn Yong Hee Hong Dong-Hwan Lee Hoon Kook Han Hyuk Lim Kyung Hee Kim Woo-Shik Kim Geu-Ru Hong Su-Hyun Kim Sang Hyun Park Chan-Duck Kim So Mi Kim Jeong-Sook Seo Han-Wook Yoo

Fabry disease is a rare X-linked lysosomal storage disorder caused by an α-galactosidase A deficiency. The progressive accumulation of globotriaosylceramide (GL-3) results in life-threatening complications, including renal, cardiac, and cerebrovascular diseases. This study investigated the phenotypic and molecular spectra of GLA mutations in Korean patients with Fabry disease using a nationwide...

Journal: :QJM : monthly journal of the Association of Physicians 2010
A Mehta M Beck F Eyskens C Feliciani I Kantola U Ramaswami A Rolfs A Rivera S Waldek D P Germain

Fabry disease is an X-linked inherited condition due to the absence or reduction of alpha-galactosidase activity in lysosomes, that results in accumulation of globotriaosylceramide (Gb3) and related neutral glycosphingolipids. Manifestations of Fabry disease include serious and progressive impairment of renal and cardiac function. In addition, patients experience pain, gastrointestinal disturba...

2017
Hernán Trimarchi Romina Canzonieri Amalia Schiel Juan Politei Cristian Costales-Collaguazo Aníbal Stern Matías Paulero Tatiana Rengel Lara Valiño-Rivas Mariano Forrester Fernando Lombi Vanesa Pomeranz Romina Iriarte Alexis Muryan Alberto Ortiz María Dolores Sanchez-Niño Elsa Zotta

Background. Despite enzyme replacement therapy, Fabry nephropathy still progresses. Podocyturia is an irreversible event that antedates proteinuria and leads to chronic renal failure. We evaluated a potential mechanism of podocyte detachment via the expression of the urokinase-type Plasminogen Activator Receptor (uPAR) in urinary podocytes of Fabry patients. Methods. This is a cross-sectional s...

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