Paradoxical Response to Enzyme Replacement Therapy of Fabry Disease Cardiomyopathy

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Paradoxical Response to Enzyme Replacement Therapy of Fabry Disease Cardiomyopathy.

A 53-year-old asymptomatic man with no family history of Fabry disease or hypertrophic cardiomyopathy (HCM) exhibited increased ECG voltages (Figure [A]) and primary cardiac hypertrophy (left ventricular maximal wall thickness 16 mm and myocardial mass 163.2 g) with preserved contractility at cardiac magnetic resonance (Figure [D]). He was diagnosed in 2006 to be affected by Fabry disease cardi...

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Cardiomyopathy and Response to Enzyme Replacement Therapy in a Male Mouse Model for Fabry Disease

Fabry disease is an X-linked disorder of glycosphingolipid metabolism that results in progressive accumulation of neutral glycosphingolipids, (predominately globotriaosylceramide; GL-3) in lysosomes, as well as other cellular compartments and the extracellular space. Our aim was to characterize the cardiac phenotype of male knock-out mice that are deficient in alpha-galactosidase A activity, as...

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Enzyme replacement therapy for Anderson-Fabry disease.

BACKGROUND Anderson-Fabry disease is an X-linked defect of glycosphingolipid metabolism. Progressive renal insufficiency is a major source of morbidity, additional complications result from cardio- and cerebro-vascular involvement. Survival is reduced among affected males and symptomatic female carriers. OBJECTIVES To evaluate the effectiveness and safety of enzyme replacement therapy compare...

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Anderson-Fabry disease: enzyme replacement therapy.

Sir, Anderson-Fabry disease is a metabolic lysosomal storage disease caused by a deficiency of the enzyme a-galactosidase A and inherited as an X-linked recessive trait. The progressive accumulation of glycosphingolipids (globotriaosylceramide, GB3) in blood, vessels and cells from several organs and tissues causes significant multi-systemic damage in homozygous males and in carrier females. Fo...

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Progression of Fabry cardiomyopathy despite enzyme replacement therapy.

Maurizio Pieroni, Antonia Camporeale, Roberta Della Bona, Alessandra Sabini, Deborah Progression of Fabry Cardiomyopathy Despite Enzyme Replacement Therapy Print ISSN: 0009-7322. Online ISSN: 1524-4539 Copyright © 2013 American Heart Association, Inc. All rights reserved. is published by the American Heart Association, 7272 Greenville Avenue, Dallas, TX 75231 Circulation doi: 10.1161/CIRCULATIO...

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ژورنال

عنوان ژورنال: Circulation: Cardiovascular Imaging

سال: 2016

ISSN: 1941-9651,1942-0080

DOI: 10.1161/circimaging.116.005078