Clinical Trials for Patients with Urea Cycle Disorders
Home About HPN-100 About UCD Who should volunteer? About the Studies Participating Centers Complete safety information for BUPHENYL^®	About Urea Cycle Disorders A urea cycle disorder (UCD) is a genetic disorder caused by a deficiency of one of the enzymes in the urea cycle which is responsible for removing ammonia from the blood stream. The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is removed from the blood and converted to urea. Normally, urea is excreted in the urine and thereby removes waste nitrogen from the body. In urea cycle disorders, the nitrogen accumulates in the form of ammonia, a highly toxic substance, and results in high levels of ammonia in the blood (hyperammonemia). Ammonia then reaches the brain through the blood, where it can cause irreversible brain damage, coma and/or death. This information is adapted from The National Urea Cycle Disorders Foundation (NUCDF), a nonprofit organization dedicated to the identification, treatment and cure of urea cycle disorders. For additional information about UCDs, please contact the NUCDF directly and/or The Urea Cycle Disorders Consortium (UCDC) of the Rare Diseases Clinical Research Network, a National Institutes of Health (NIH)-funded research initiative.
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