American patient organisation founded in 1988 and based in California, devoted to urea cycle disorders, a group of inherited metabolic diseases in which ammonia builds up in the blood. Supports patients and families with information, a helpline and family conferences, funds and helps organise research including the Urea Cycle Disorders Consortium and patient registries, works with clinicians and drug developers on nitrogen scavenging medicines, liver transplantation and gene therapy trials, and campaigns for newborn screening and access to treatment.
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