Plasma and urinary NMR metabolic profiling of the phenylalanine-tyrosine pathway in nitisinone-treated alkaptonuria: comparison of two dosing regimens and identification of a candidate pharmacodynamic biomarker.

Orfadin (nitisinone) - Astellas, SOBI

http://www.ncbi.nlm.nih.gov/pubmed/42685651

Andrea Bernini; Andrea Mazzon; Annalisa Santucci; Bruno Frediani; Daniela Grasso; Elena Bianchi; Gabriella Iacomelli; Luana Peruzzi; Marco Bardelli; Valentina Balloni

Sep 3, 2026