![]() Teckman says those impacted by the disease are often undiagnosed or misdiagnosed as fatty liver disease, asthma, or smoking-related lung disease. Symptoms may include shortness of breath and wheezing, repeated infections of the lungs, yellow skin, fatigue, cirrhosis of the liver, liver failure and even death. Teckman is a leading authority on AAT deficiency, which affects 1 in 3,500 births and causes severe lung disease in adults or liver disease in adults and children. “We have patients come around the country to see SLU’s expert faculty members at SSM Health Cardinal Glennon Children’s Hospital with this disease for care and to participate in our studies.” “This is the culmination of over a decade of work to cure this disease, and a significant part of the work was done here,” said Teckman, who also is director of pediatric gastroenterology and hepatology at SLU. Jeffrey Teckman, M.D., professor of pediatrics and biochemistry and molecular biology, is the paper's senior author. AAT is a protein made in the liver and released into the blood in large quantities to help protect the body when warding off infections. The multicenter, phase 2, open-label trial investigated the safety and efficacy of fazirsiran, an RNA interference drug, in patients 18 to 75 years of age with liver disease associated with alpha-1 antitrypsin (AAT) deficiency. ![]() The study, “ Fazirsiran for Liver Disease Associated with Alpha1-Antitrypsin Deficiency,” was published online in the New England Journal of Medicine, one of the world’s leading medical journals. LOUIS - Researchers at Saint Louis University's School of Medicine, in collaboration with Arrowhead Pharmaceuticals and Takeda Pharmaceuticals, report the first effective drug to treat a rare, genetic liver disease that formerly could only be treated with a liver transplant. Image: Jeffrey Teckman, M.D., professor of pediatrics and biochemistry and molecular biology at Saint Louis University School of Medicine. ![]()
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