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Researchers find drug combo to treat cystic fibrosis

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Researchers at Queen’s University, Belfast, and St Vincent’s Hospital Dublin have identified a combination of three drugs to treat the underlying cause of the cystic fibrosis. 

The drug targets the root cause of cystic fibrosis, a genetic condition which clogs up the lungs and digestive system, making breathing difficult resulting in an early death for those affected.

Cystic fibrosis (CF) is a genetic condition affecting more than 10,400 people in the UK.  It is a progressive and life-threatening disease which results in the formation of thick mucus that builds up in the lungs and other parts of the body. It leads to severe respiratory infections that damage the lungs and cause them to fail.

CF is caused by a defective protein that results from mutations in the CFTR gene. The most common mutation is the F508del mutation. Although there is currently no cure for CF, it is possible to help control the symptoms and delay complications to make the condition easier to live with.

“The trial was a success in demonstrating that this drug combination can potentially treat up to 90% of people with CF by addressing the underlying cause of their disease. This new “triple therapy” has the potential to transform the lives of people with CF. It results in a significant improvement in lung function and quality of life and also reduces the frequency of chest infections. This treatment will likely alter the future of CF care.” Said Dr. Damian Downey Clinical Senior Lecturer at the Wellcome-Wolfson Institute for Experimental Medicine, Queen’s University Belfast

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