TY  - JOUR 
AU  - Spiekerkoetter, Ute
AU  - Couce Pico, María Luz
AU  - Das, Anibh M
AU  - de Laet, Corinne
AU  - Lund, Allan M
AU  - Dionisi-Vici, Carlo
AU  - Schiff, Manuel
AU  - Spada, Marco
AU  - Sparve, Erik
AU  - Szamosi, Johan
AU  - Vara, Roshni
AU  - Rudebeck, Mattias
PY  - 2021
SN  - 2213-8587
UR  - http://hdl.handle.net/20.500.11940/22270
AB  - [EN] Findings 315 patients were enrolled during the study period (complete set). Additionally, data from 24 patients who had liver transplantation or died during the post-marketing surveillance programme were retrieved (extended analysis set; 339...
LA  - eng
KW  - Pregnancy
KW  - Tyrosinemias
KW  - Enzyme Inhibitors
KW  - Safety
KW  - Adolescent Medicine
KW  - Cyclohexanones
KW  - Neonatal Screening
KW  - Nitrobenzoates
KW  - Kidney Diseases
KW  - Life
KW  - Tyrosine
TI  - Long-term safety and outcomes in hereditary tyrosinaemia type 1 with nitisinone treatment: a 15-year non-interventional, multicentre study
DO  - 10.1016/S2213-8587(21)00092-9
T2  - Lancet Diabetes Endocrinology
M2  - 427
KW  - IDIS
KW  - CHUS
VL  - 9
ER  -