Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1

Tyrosinemia type 1

Taste and Palatability of Orfadin Suspension Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin Bio Equivalency 20 Mgm Orfadin and 20 Mgm of Nitisonine Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1) Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1) Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 Biomarker for the Early Diagnosis and Monitoring in Tyrosinemia Type 1 (BioTyrosin) Hereditary Hepatorenal Tyrosinemia Natural History in Egypt and the Arab World (Multicenter Clinical Study) Phase II Study of the Enzyme Inhibitor NTBC for Tyrosinemia Type I Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care Evaluation of TYR Sphere