STANFORD, Calif. -- People with a class of rare genetic disorders that often lead to brain damage, coma and death can be successfully treated with drugs, says a researcher at the Stanford University School of Medicine and Lucile Packard Children’s Hospital.The researchers found in their unprecedented 25-year study that prompt diagnosis coupled with a rapid start of intravenous drug therapy significantly improves the survival rates of people with the condition, called urea cycle disorders. The condition affects proteins in the liver that are necessary to process the by-products of protein metabolism.
