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Open-Label Single-Sequence Crossover Study Evaluating Pharmacokinetics, Efficacy, and Safety of Once-Daily Dosing of Nitisinone in Patients with Hereditary Tyrosinemia Type 1

Background: Although nitisinone is successfully used to treat hereditary tyrosinemia type 1 (HT-1) with the recommended twice-daily dosing, data describing a long half-life motivate less frequent dosing. Therefore, in agreement with the Pharmacovigilance Risk Assessment Committee at the European Med...

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Detalhes bibliográficos
Publicado no:JIMD Rep
Main Authors: Guffon, Nathalie, Bröijersén, Anders, Palmgren, Ingrid, Rudebeck, Mattias, Olsson, Birgitta
Formato: Artigo
Idioma:Inglês
Publicado em: Springer Berlin Heidelberg 2017
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5874213/
https://ncbi.nlm.nih.gov/pubmed/28643275
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2017_29
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