Evaluation of pre-symptomatic nitisinone treatment on long-term outcomes in Tyrosinemia type 1 patients: a systematic review

BACKGROUND: Tyrosinemia type 1 (TYR1) is a rare autosomal recessive disorder of amino acid metabolism that is fatal without treatment. With medication (nitisinone) and dietary restrictions outcomes are improved. We conducted a systematic review to investigate if treatment with nitisinone following s...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Geppert, Julia, Stinton, Chris, Freeman, Karoline, Fraser, Hannah, Clarke, Aileen, Johnson, Samantha, Sutcliffe, Paul, Taylor-Phillips, Sian
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2017
Assuntos:
Review
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5594482/
https://ncbi.nlm.nih.gov/pubmed/28893311
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-017-0696-z
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