What is a Urea Cycle Disorder?
A urea cycle disorder, also known as UCD, is a rare genetic condition in which one of the enzymes in the urea cycle is missing. These enzymes are responsible for removing ammonia that generates from ingestion of protein from the blood. In UCD, nitrogen, a waste product of protein metabolism, accumulates in the form of ammonia. Elevated blood ammonia, or hyperammonemia, can cause irreversible brain damage, coma and or death.
What is THRIVE?
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years. As an observational study, enrolled patients will not be required to make any additional office visits or take any medicine outside of normal care.
By allowing their information to be collected in the study, patients may help doctors and researchers better understand how different medicines affect individuals with UCDs, and help doctors take care of patients with UCDs in the future.
Who can participate in THRIVE?
Patients may qualify to participate in THRIVE if they have a confirmed or suspected diagnosis of a UCD. The doctor will explain the study, and will ask patients to sign an Informed Consent or Assent Form to join THRIVE.
Talk to your doctor to learn more about THRIVE.