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WASHINGTON, DC-Parents of children born with tyrosinemia were warned that those inflicted with the hereditary disease have a 29% survival rate. The rare liver disease prevents the body from properly breaking down specific amino acids.
However, a rare solution may help fight the rare disease. Researchers at Sweden's Gothenberg University noticed a failed herbicide, produced by Zeneca Inc., broke down the same amino acid compounds. The drug Orfadin was created from the failed weed killer, and patented by a mall Nashville-based pharmaceutical company-Rare Disease Therapeutics.
Hereditary tyrosinemia type 1 causes progressive liver failure and liver cancer in young children. Yet in Orfadin studies, 89% of those inflicted lived four years or more. The drug blocks specific liver-destroying proteins. Previous treatments centered on no-protein diets, but were rarely successful.
The drug will cost $12,000 per year for infants, but Rare Disease Therapeutic vice president Bo Allen has said no child will be turned away from treatment.
The company received a special seven-year patent on the drug through an FDA program for rare diseases.
There are an estimated 100 children with the disease.
Information from www.msnbc.com