نتایج جستجو برای: fabry disease

تعداد نتایج: 1493456  

Journal: :Archives of clinical neuropsychology : the official journal of the National Academy of Neuropsychologists 2014
Linda Sigmundsdottir Michel C Tchan Alex A Knopman Graham C Menzies Jennifer Batchelor David O Sillence

Fabry disease is an X-linked lysosomal storage disorder which can result in renal, cardiac, and cerebrovascular disease. Patients are at increased risk of stroke and neuroimaging studies note cerebrovascular pathology. This study provides a cognitive profile of a cohort of individuals with Fabry disease and investigates the impact of pain, age, renal, cardiac, and cerebrovascular functioning on...

2014
Nurcan Üçeyler György A. Homola Hans Guerrero González Daniela Kramer Christoph Wanner Frank Weidemann László Solymosi Claudia Sommer

A high load of white matter lesions and enlarged basilar arteries have been shown in selected patients with Fabry disease, a disorder associated with an increased stroke risk. We studied a large cohort of patients with Fabry disease to differentially investigate white matter lesion load and cerebral artery diameters. We retrospectively analyzed cranial magnetic resonance imaging scans of 87 con...

Journal: :Acta dermatovenerologica Croatica : ADC 2006
Branka Marinović

More than a hundred years ago, in 1898, two dermatologists, William Anderson in England and Johannes Fabry in Germany, independently reported on patients with multiple angiokeratomas as well as some other symptoms. Today, the disease is called Anderson-Fabry or only Fabry disease, or angiokeratoma corporis diffusum, the latter being more often found in dermatologic literature. Fabry disease is ...

2013
Eric Alamartine Aurore Sury Frédéric Roche Vincent Pichot Jean-Claude Barthelemy

Background: Fabry disease is an inherited, multisystemic and progressive lysosomal storage disorder. The first symptoms of Fabry neuropathy reflect progressive loss of function of both peripheral somatic and autonomic nerve cells. We aimed to evaluate autonomic nervous system (ANS) activity in a cohort of patients with Fabry disease. Methods: ANS activity was evaluated by determining heart rate...

2015
Susanne Pitz Gisela Kalkum Laila Arash Nesrin Karabul Andrea Sodi Sylvain Larroque Michael Beck Andreas Gal

Ocular signs in Fabry disease have generally been regarded to be primarily of diagnostic value. We explored whether ocular findings, alone or in particular in combination with the α-galactosidase A gene mutation, have predictive value for disease severity. Data from the Fabry Outcome Survey (FOS), a large, global database sponsored by Shire, were selected for adult patients who had undergone op...

Journal: :Journal of Inborn Errors of Metabolism and Screening 2016

2004
Maria Fuller Melanie Lovejoy Doug A. Brooks Miriam L. Harkin John J. Hopwood Peter J. Meikle

Background: Fabry disease is an X-linked inborn error of glycosphingolipid catabolism resulting from a deficiency of the lysosomal exoglycohydrolase, -galactosidase. Enzyme replacement therapy is currently available for Fabry disease, but early diagnosis before the onset of irreversible pathology will be mandatory for successful treatment. Presymptomatic detection would be possible through the ...

2013
Hsiang-Yu Lin Hao-Chuan Liu Yu-Hsiu Huang Hsuan-Chieh Liao Ting-Rong Hsu Chia-I Shen Shao-Tzu Li Cheng-Fang Li Li-Hong Lee Pi-Chang Lee Chun-Kai Huang Chuan-Chi Chiang Ching-Yuang Lin Shuan-Pei Lin Dau-Ming Niu

OBJECTIVE Current studies of newborn screening for Fabry disease in Taiwan have revealed a remarkably high prevalence of cardiac-type Fabry disease with a Chinese hotspot late-onset Fabry mutation (IVS4+919G>A). DESIGN Retrospective cohort study. SETTING Tertiary medical centre. PARTICIPANTS 21 patients with cardiac-type Fabry disease (15 men and 6 women) as well as 15 patients with class...

Journal: :PEDIATRICS 2005

Journal: :International journal of molecular medicine 2009
Eun-Sook Park Jin-Ok Choi Joo-Won Park Mi Hee Lee Hae-Young Park Sung-Chul Jung

Fabry disease is a lysosomal storage disease caused by a deficiency of alpha-galactosidase A, which results in aberrant glycosphingolipid metabolism and accumulation of globotriaosylceramide (Gb3). Since a correlation between the level of Gb3 and clinical manifestations of Fabry disease has not been observed, we investigated potential diagnostic biomarkers. Hepatic and renal gene expression of ...

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