Literature DB >> 23756328

Acitretin-responsive ichthyosis in Chanarin-Dorfman syndrome with a novel mutation in the ABHD5/CGI-58 gene.

Rangan Srinivasaraghavan1, Sriram Krishnamurthy, Rumesh Chandar, Denise Cassandrini, Subramanian Mahadevan, Claudio Bruno, Filippo M Santorelli.   

Abstract

Chanarin-Dorfman syndrome (CDS) is a rare nonlysosomal neutral lipid storage disorder characterized by congenital ichthyosis, lipid vacuoles in leukocytes (Jordan's anomaly), and hepatomegaly. The authors herein report an 18-month-old boy with ichthyosis and hepatomegaly diagnosed with CDS and confirmed to have a novel c.506-3C>G mutation in the ABHD5/CGI-58 gene. Our case also illustrates that retinoids such as acitretin could be useful in the treatment of skin manifestations in CDS even in the presence of liver derangement.
© 2013 Wiley Periodicals, Inc.

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Year:  2013        PMID: 23756328     DOI: 10.1111/pde.12170

Source DB:  PubMed          Journal:  Pediatr Dermatol        ISSN: 0736-8046            Impact factor:   1.588


  4 in total

1.  Dorfman-Chanarin Syndrome: A Rare Cause of Metabolic Associated Fatty Liver Disease Related to Homozygosity of the Nonsense Mutation c.934C>T (p.R312*).

Authors:  Rita Quelhas da Costa; Francisco Laranjeira; Isaura Duarte Ribeiro; António Filipe Santos; Filipe Nery
Journal:  GE Port J Gastroenterol       Date:  2021-07-07

Review 2.  Inborn errors of cytoplasmic triglyceride metabolism.

Authors:  Jiang Wei Wu; Hao Yang; Shu Pei Wang; Krishnakant G Soni; Catherine Brunel-Guitton; Grant A Mitchell
Journal:  J Inherit Metab Dis       Date:  2014-10-10       Impact factor: 4.982

3.  Targeted Therapies for Metabolic Myopathies Related to Glycogen Storage and Lipid Metabolism: a Systematic Review and Steps Towards a 'Treatabolome'.

Authors:  A Manta; S Spendiff; H Lochmüller; R Thompson
Journal:  J Neuromuscul Dis       Date:  2021

4.  Recurrent N209* ABHD5 mutation in two unreported families with Chanarin Dorfman Syndrome.

Authors:  Daniela Tavian; Murat Durdu; Corrado Angelini; Enza Torre; Sara Missaglia
Journal:  Eur J Transl Myol       Date:  2021-05-12
  4 in total

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