Type 1 tyrosinemia in Finland: a nationwide study

Type 1 tyrosinemia in Finland: a nationwide study

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 normalizat...

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Bibliographic Details
Main Authors: Linnea Äärelä, Pauliina Hiltunen, Tea Soini, Nina Vuorela, Heini Huhtala, Pasi I. Nevalainen, Markku Heikinheimo, Laura Kivelä, Kalle Kurppa
Format: Article
Language:English
Published: BMC 2020-10-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Tyrosinemia
Succinylacetone
Liver transplant
Nitisinone
Screening
Online Access:http://link.springer.com/article/10.1186/s13023-020-01547-w

Internet

http://link.springer.com/article/10.1186/s13023-020-01547-w

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