Renal involvement in Fabry disease

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Renal involvement in Fabry disease.

Every cell in the human body has globotriaosylceramide accumulation (Gb3) in Fabry disease due to the mutation in gene of the enzyme α-galactosidase A. It is a disease linked to sex. The main clinical features are: cutaneous angiokeratomas; acroparestesias and early strokes; decreased sweating and heat intolerance; ocular changes; myocardial hypertrophy, arrhythmias; gastrointestinal disorders ...

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Early Renal Involvement in a Girl with Classic Fabry Disease

Fabry disease is an X-linked lysosomal storage disorder resulting from the deficiency or absence of the enzyme alpha galactosidase A; this defect leads to the systemic accumulation of globotriaosylceramide and its metabolites. Organic involvement in men is well known, but in women it is controversial, mainly due to the random X-chromosome inactivation in each of their cells (Lyon hypothesis). T...

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Motor involvement in Fabry disease

We have read with great interest the article published by Wise and colleagues on the last issue of Molecular Genetics and Metabolism Reports [1]. The Authors performed a survey study investigating the prevalence of Parkinson Disease (PD) in individuals affected by Fabry Disease (FD), and found that 2 patients out of 90 (2.2%) were diagnosed with PD. Although considering all the limitations of t...

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Renal pathology in Fabry disease.

JOSEPH ALROY,* SHARDA SABNIS, and JEFFREY B. KOPP *Department of Pathology, Tufts University Schools of Veterinary Medicine and Medicine, and New England Medical Center, Boston, Massachusetts; Division of Nephrology, Armed Forces Institute of Pathology, Washington, DC; and Kidney Disease Section, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Be...

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Bone and joint involvement in Fabry disease.

Fabry disease (FD) is an X-linked lysosomal storage disease caused by deficient activity of the enzyme alpha-galactosidase A. Although the disease has progressive effects on most organ systems in the body, data is limited regarding skeletal involvement in this rare disorder. We describe four family-related patients, three men and one premenopausal female, sharing a classic phenotype of FD. Dual...

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ژورنال

عنوان ژورنال: Jornal Brasileiro de Nefrologia

سال: 2016

ISSN: 0101-2800

DOI: 10.5935/0101-2800.20160034