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Nitisinone for the treatment of hereditary tyrosinemia type I
Expert Opinion on Orphan Drugs, 2013Introduction: Nitisinone has transformed the management of hereditary tyrosinemia type 1 (HT1) and if combined with neonatal screening could abolish most of the clinical manifestations of the disease. Hereditary tyrosinemia type 1 is a rare genetic disease due to fumarylacetoacetase (FAH) deficiency, which usually presents with liver failure.
openaire +1 more source
Liver transplantation for tyrosinemia in the nitisinone era
2019[No abstract available]
Karaca, C. +5 more
openaire +1 more source
First Scandinavian case of successful pregnancy during nitisinone treatment for type 1 tyrosinemia
Journal of Pediatric Endocrinology and Metabolism, 2020Pasi Nevalainen +2 more
exaly
Experience of nitisinone for the pharmacological treatment of hereditary tyrosinaemia type 1
Expert Opinion on Pharmacotherapy, 2008Saikat Santra
exaly
LC-MS/MS study of the degradation processes of nitisinone and its by-products
Journal of Pharmaceutical and Biomedical Analysis, 2019Rafal Rola +2 more
exaly