Literature DB >> 20493597

[Type B Niemann Pick disease: clinical description of three patients in a same family].

C Alizon1, A-B Beucher, A-L Gourdier, C Lavigne.   

Abstract

INTRODUCTION: The Niemann Pick disease type B is a rare deficiency in sphingomyelinase activity, autosomal recessively inherited. CASE REPORTS: We report three patients (two men, one woman) of the same family, who showed pulmonary and hepatosplenic lesions, usually present in the disease but also adrenal gland lesions confirmed by tomodensitometry.
CONCLUSION: The current treatment of Niemann Pick disease is purely symptomatic awaiting the use of enzymatic replacement therapy which has been successfully experimented in animal model. Copyright (c) 2010 Société nationale française de médecine interne (SNFMI). Published by Elsevier SAS. All rights reserved.

Entities:  

Mesh:

Year:  2010        PMID: 20493597     DOI: 10.1016/j.revmed.2010.01.009

Source DB:  PubMed          Journal:  Rev Med Interne        ISSN: 0248-8663            Impact factor:   0.728


  3 in total

Review 1.  An overview of inborn errors of metabolism manifesting with primary adrenal insufficiency.

Authors:  Fady Hannah-Shmouni; Constantine A Stratakis
Journal:  Rev Endocr Metab Disord       Date:  2018-03       Impact factor: 6.514

2.  Coincidence of Niemann-Pick Disease and beta-Thalassemia; a Case Report.

Authors:  Fatemeh Farahmand; Vajiheh Modaresi; Mina Izadyar; Fatemeh Mahjob
Journal:  Iran J Pediatr       Date:  2010-12       Impact factor: 0.364

3.  New Single Nucleotide Deletion In the SMPD1 Gene Causes Niemann Pick Disease Type A in a Child from Southwest Iran: A Case Report.

Authors:  Hamid Galehdari; Raheleh Tangestani; Sepideh Ghasemian
Journal:  Iran J Pediatr       Date:  2013-04       Impact factor: 0.364

  3 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.