2020
DOI: 10.21203/rs.3.rs-40438/v1
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Clinical presentation and long-term outcomes of type 1 tyrosinemia in Finland: a nationwide study

Abstract: Abstract Background: Introduction of nitisinone and newborn screening (NBS) have transformed the treatment of type 1 tyrosinemia, but the effects of these changes on the long-term outcomes remain obscure. Also, the predictors for later complications, the significance of drug levels and the normalization of laboratory and imaging findings are poorly known. We investigated these issues in a nationwide study. Results: Type 1 ty… Show more

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