Literature DB >> 8901188

[Albinism, thrombopathy, ceroid storage disease--Hermansky-Pudlak syndrome. Overview and description with immunodeficiency].

F Berz1, M Weiss, B H Belohradsky.   

Abstract

The Hermansky-Pudlak Syndrome (HPS) is a rare autosomal recessively inherited triad of oculocutaneous albinism hemorrhagic diathesis and accumulation of ceroid in tissues. This article comprising the published reports of 232 patients is extended by the personal observation of a 7-year-old boy with HPS and immune deficiency due to a reduced activity of "natural killer" cells. In most cases the syndrome belongs to a form of tyrosinase-positive albinism with great clinical variability. The prolonged bleeding time is due to the lack of storage organelles (dense bodies) in thrombocytes. The most frequent complication of ceroid storage is fibrotic, restrictive lung disease. So far no curative therapy exists; the nature of the defect is still unknown.

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Year:  1996        PMID: 8901188     DOI: 10.1055/s-2008-1044000

Source DB:  PubMed          Journal:  Klin Padiatr        ISSN: 0300-8630            Impact factor:   1.349


  1 in total

1.  A Novel Splice Site Mutation in HPS1 Gene is Associated with Hermansky-Pudlak Syndrome-1 (HPS1) in an Iranian Family.

Authors:  Soudeh Ghafouri-Fard; Feyzollah Hashemi-Gorji; Vahid Reza Yassaee; Nasrin Alipour; Mohammad Miryounesi
Journal:  Int J Mol Cell Med       Date:  2016-07-03
  1 in total

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