Literature DB >> 27061865

Multiple parapelvic cysts in Fabry disease.

María A Azancot1, Josefa Vila1, Carmen Domínguez2, Xavier Serres3, Eugenia Espinel4.   

Abstract

Fabry disease is an inherited, X-linked lysosomal storage disorder caused by deficiency of the enzyme alpha galactosidase A (alpha-GLA A), which leads to glycosphingolipid accumulation, mainly globotriaosylceramide, in tissues. Disease prevalence and the index of suspicion are both low, which tends to result in delayed diagnosis and treatment. We present the case of a male Fabry disease patient who manifested no angiokeratoma lesions but presented multiple parapelvic cysts and renal failure. The genetic study revealed an alpha-GLA A gene mutation that had not been recorded in the mutations registry. The de novo mutation was not found in his relatives and it was not transmitted to his offspring. The large number and peculiar appearance of the parapelvic cysts led to the diagnosis.
Copyright © 2016 Sociedad Española de Nefrología. Published by Elsevier España, S.L.U. All rights reserved.

Entities:  

Keywords:  De novo mutation; Enfermedad de Fabry; Fabry disease; Mutación de novo; Parapelvic cysts; Quistes parapiélicos

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Year:  2016        PMID: 27061865     DOI: 10.1016/j.nefro.2015.12.003

Source DB:  PubMed          Journal:  Nefrologia        ISSN: 0211-6995            Impact factor:   2.033


  1 in total

1.  Fabry disease in the Spanish population: observational study with detection of 77 patients.

Authors:  Irene Vieitez; Olga Souto-Rodriguez; Lorena Fernandez-Mosquera; Beatriz San Millan; Susana Teijeira; Julian Fernandez-Martin; Felisa Martinez-Sanchez; Luis Jose Aldamiz-Echevarria; Monica Lopez-Rodriguez; Carmen Navarro; Saida Ortolano
Journal:  Orphanet J Rare Dis       Date:  2018-04-10       Impact factor: 4.123

  1 in total

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