Literature DB >> 25646736

Radiographic features of the skeleton in disorders of post-squalene cholesterol biosynthesis.

Massimiliano Rossi1, Christine M Hall, Raymonde Bouvier, Sophie Collardeau-Frachon, Frédérique Le Breton, Martine Bucourt, Marie Pierre Cordier, Christine Vianey-Saban, Giancarlo Parenti, Generoso Andria, Martine Le Merrer, Patrick Edery, Amaka C Offiah.   

Abstract

Disorders of post-squalene cholesterol biosynthesis are inborn errors of metabolism characterised by multiple congenital abnormalities, including significant skeletal involvement. The most frequent and best-characterised example is the Smith-Lemli-Opitz syndrome. Nine other disorders are known, namely autosomal-recessive Antley-Bixler syndrome, Greenberg dysplasia, X-linked dominant chondrodysplasia punctata, X-linked recessive male emopamil-binding protein deficiency, CHILD syndrome, CK syndrome, sterol C4 methyloxidase-like deficiency, desmosterolosis and lathosterolosis. This study provides an overview of the radiologic features observed in these diseases. A common pattern of limb abnormalities is recognisable, including polydactyly, which is typically post-axial and rarely interdigital and can involve all four limbs, and syndactyly of the toes. Chondrodysplasia punctata is specifically associated with a subgroup of disorders of cholesterol biosynthesis (Greenberg dysplasia, CHILD syndrome, X-linked dominant chondrodysplasia punctata, male emopamil-binding protein deficiency). The possible occurrence of epiphyseal stippling in the Smith-Lemli-Opitz syndrome, initially reported, does not appear to be confirmed. Stippling is also associated with other congenital disorders such as chromosomal abnormalities, brachytelephalangic chondrodysplasia punctata (X-linked recessive chondrodysplasia punctata, disruptions of vitamin K metabolism, maternal autoimmune diseases), rhizomelic chondrodysplasia punctata (peroxisomal disorders) and lysosomal storage disorders. In the differential diagnosis of epiphyseal stippling, a moth-eaten appearance of bones, asymmetry, or presence of a common pattern of limb abnormalities indicate inborn errors of cholesterol biosynthesis. We highlight the specific differentiating radiologic features of disorders of post-squalene cholesterol biosynthesis.

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Year:  2015        PMID: 25646736     DOI: 10.1007/s00247-014-3257-9

Source DB:  PubMed          Journal:  Pediatr Radiol        ISSN: 0301-0449


  69 in total

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2.  Mutations in a delta 8-delta 7 sterol isomerase in the tattered mouse and X-linked dominant chondrodysplasia punctata. jderry@immunex.com.

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Journal:  Am J Hum Genet       Date:  2010-12-10       Impact factor: 11.025

4.  HEM dysplasia and ichthyosis are likely laminopathies and not due to 3beta-hydroxysterol Delta14-reductase deficiency.

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5.  Clinical phenotype of desmosterolosis.

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Journal:  Am J Med Genet       Date:  1998-01-13

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Authors:  Hans R Waterham; Janet Koster; Petra Mooyer; Gerard van Noort Gv; Richard I Kelley; William R Wilcox; Ronald J A Wanders; Raoul C M Hennekam; Jan C Oosterwijk
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9.  Two novel EBP mutations in Conradi-Hünermann-Happle syndrome.

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Journal:  Eur J Med Genet       Date:  2007-06-03       Impact factor: 2.708

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Review 2.  Antenatal manifestations of inborn errors of metabolism: autopsy findings suggestive of a metabolic disorder.

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4.  Zebrafish models of skeletal dysplasia induced by cholesterol biosynthesis deficiency.

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6.  Intracellular biosynthesis of lipids and cholesterol by Scap and Insig in mesenchymal cells regulates long bone growth and chondrocyte homeostasis.

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