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Type 1 tyrosinemia in Finland: a nationwide study
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 l...
Autores principales: | Äärelä, Linnea, Hiltunen, Pauliina, Soini, Tea, Vuorela, Nina, Huhtala, Heini, Nevalainen, Pasi I., Heikinheimo, Markku, Kivelä, Laura, Kurppa, Kalle |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7549233/ https://www.ncbi.nlm.nih.gov/pubmed/33046095 http://dx.doi.org/10.1186/s13023-020-01547-w |
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