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From genotype to phenotype: Early prediction of disease severity in argininosuccinic aciduria

Argininosuccinic aciduria (ASA) is an inherited urea cycle disorder and has a highly variable phenotypic spectrum ranging from individuals with lethal hyperammonemic encephalopathy, liver dysfunction and cognitive deterioration, to individuals with a mild disease course. Since it is difficult to pre...

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Bibliografske podrobnosti
izdano v:Hum Mutat
Main Authors: Zielonka, Matthias, Garbade, Sven F., Gleich, Florian, Okun, Jürgen G., Nagamani, Sandesh C. S., Gropman, Andrea L., Hoffmann, Georg F., Kölker, Stefan, Posset, Roland
Format: Artigo
Jezik:Inglês
Izdano: 2020
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC7428858/
https://ncbi.nlm.nih.gov/pubmed/31943503
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/humu.23983
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