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Natural history of cerebrotendinous xanthomatosis: a paediatric disease diagnosed in adulthood

Cerebrotendinous xanthomatosis (CTX) is among the few inherited neurometabolic disorders amenable to specific treatment. It is easily diagnosed using plasma cholestanol. We wished to delineate the natural history of the most common neurological and non-neurological symptoms in thirteen patients with...

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Podrobná bibliografie
Vydáno v:Orphanet J Rare Dis
Hlavní autoři: Degos, Bertrand, Nadjar, Yann, Amador, Maria del Mar, Lamari, Foudil, Sedel, Frédéric, Roze, Emmanuel, Couvert, Philippe, Mochel, Fanny
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2016
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4833925/
https://ncbi.nlm.nih.gov/pubmed/27084087
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-016-0419-x
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