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Cerebrotendinous Xanthomatosis: diversity of presentation and refining treatment with chenodeoxycholic acid

BACKGROUND: Cerebrotendinous xanthomatosis (CTX) is a rare but treatable neurometabolic disorder of lipid storage and bile acid synthesis. Whilst CTX is said to present with the classic triad of juvenile onset cataracts, tendon xanthomata and progressive ataxia, the diversity of presentation can be...

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Detaylı Bibliyografya
Yayımlandı:Cerebellum Ataxias
Asıl Yazarlar: Islam, Mahjabin, Hoggard, Nigel, Hadjivassiliou, Marios
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: BioMed Central 2021
Konular:
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC7844909/
https://ncbi.nlm.nih.gov/pubmed/33509302
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s40673-021-00128-2
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