Baylor College of Medicine and the National Urea Cycle Disorders Foundation (NUCDF) have together established the CureUCD Center for Preclinical Therapeutic Discovery to advance the understanding, treatment, and cure of urea cycle disorders, which are caused by a mutation resulting in a deficiency of one of the six enzymes in the urea cycle.
The urea cycle is responsible for detoxifying ammonia, a by-product of protein metabolism, in the bloodstream. In urea cycle disorders, ammonia builds up to toxic levels that, without intervention, damage the brain and can result in coma and death.
Supported by NUCDF, which serves patients and families affected by these conditions, the CureUCD Center will engage technology and drug companies in early stage testing and validation studies of new small molecules, nucleic acids, and cell and gene therapies for these disorders.