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
Point-of-Care Engagement in Long-Term Care Decreasing Infections
November 26th 2024Get Well’s digital patient engagement platform decreases hospital-acquired infection rates by 31%, improves patient education, and fosters involvement in personalized care plans through real-time interaction tools.
Comprehensive Strategies in Wound Care: Insights From Madhavi Ponnapalli, MD
November 22nd 2024Madhavi Ponnapalli, MD, discusses effective wound care strategies, including debridement techniques, offloading modalities, appropriate dressing selection, compression therapy, and nutritional needs for optimal healing outcomes.
The Leapfrog Group and the Positive Effect on Hospital Hand Hygiene
November 21st 2024The Leapfrog Group enhances hospital safety by publicizing hand hygiene performance, improving patient safety outcomes, and significantly reducing health care-associated infections through transparent standards and monitoring initiatives.