Researchers find targeting rare form of cystic fibrosis earlier prevents organ damage in ferrets

A team of researchers from the University of Iowa, University Medical Center Utrecht and Vertex Pharmaceuticals Incorporated, reports that earlier administration of a drug already given to patients with a rare form of cystic fibrosis can prevent organ damage—at least in ferrets. In their paper published in the journal Science Translational Medicine, the group describes their study of the impact […]

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Discovery could improve cystic fibrosis treatment

Researchers exploring the effects of a long-standing treatment for cystic fibrosis have discovered a potential new target for drugs to treat the disease, which has no cure and typically cuts decades off the lives of patients. The research, a collaboration between the University of Saskatchewan in Canada and UC Berkeley, is based on a unique method developed by the Canadian […]

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Researchers identify risk factors of advanced liver disease in cystic fibrosis patients

Children’s Hospital Colorado (Children’s Colorado) pediatric gastroenterologist, Michael Narkewicz, MD, recently shared results of the Prospective Study of Ultrasound to Predict Hepatic Cirrhosis in Cystic Fibrosis (PUSH), which sought to determine if liver ultrasounds could identify children and adolescents with cystic fibrosis who are at greater risk of developing advanced liver disease. The Cystic Fibrosis Liver Disease Network PUSH study […]

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How astrophysics could transform the treatment of cystic fibrosis and other rare diseases

It’s a cruel disease which dramatically shortens life expectancy. One in 25 Europeans carry the cystic fibrosis gene and, in the UK, about 10,400 people currently have the condition. But people are living longer and longer thanks to improvements in screening at birth, early treatment and medication. One of the most important things is for patients to be put on […]

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