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Apparent underdiagnosis of Cerebrotendinous Xanthomatosis revealed by analysis of ~60,000 human exomes

Cerebrotendinous Xanthomatosis (CTX) is a treatable inborn error of metabolism caused by recessive variants in CYP27A1. Clinical presentation varies, but typically includes infant-onset chronic diarrhea, juvenile-onset bilateral cataracts, and later-onset tendinous xanthomas and progressive neurolog...

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Détails bibliographiques
Publié dans:Mol Genet Metab
Auteurs principaux: Appadurai, Vivek, DeBarber, Andrea, Chiang, Pei-Wen, Patel, Shailendra B., Steiner, Robert D, Tyler, Charles, Bonnen, Penelope E.
Format: Artigo
Langue:Inglês
Publié: 2015
Sujets:
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4767010/
https://ncbi.nlm.nih.gov/pubmed/26643207
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2015.10.010
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