The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
Homocystinuria Due to CBS Deficiency
Patients who are clinically diagnosed with homocystinuria
Male/female patients aged 1 to 65 years
Patients who consented and/or assented
Patients who are willing and able to comply with all study-related procedures.
Medically significant postnatal complications or congenital anomalies that are not associated with homocystinuria
Received any experimental therapy for homocystinuria during the 6 months prior to enrollment or expected to receive any such therapy during duration of the study
January 17, 2023
Primary Contact Information
Travere Call Center
For more information on this trial, visit clinicaltrials.gov.
For more information and to contact the study team: