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A mouse runs around in the cage; then stops to clean her white fur. She puts her pink nose up and sniffs the air. Her whiskers wiggle.
US statistics have identified there are 120,000 patients a year waiting for organ transplant in the US alone; this number is much higher worldwide. Of those 120,000, only approximately 20,000 have transplants performed. 100,000 wait patiently for the call.
The mouse runs to the wheel, her pink feet start to turn it and she hustles.
One subset of these 120,000 patients is a group of about 11,000 pediatric patients with a handful of disorders of the liver that they were born with called inborn errors of metabolism (IEM). Examples of IEM include patients with fumarylacetoacetate hydrolase (FAH), ornithine transcarbamylase (OTC) and Alpha-1 antitrypsin deficiencies. These deficiencies are hereditary, passed down from a faulty gene in each parent that pair; creating a disorder in which abnormal chemical reactions occur in the child. Treatments for metabolic liver disease may include dietary changes, phlebotomy, medications and phototherapy, but most eventually require a liver transplant; usually in adolescence.