Improving survival rates for people with cystic fibrosis

Cystic fibrosis (CF) is an inherited condition, caused by a genetic mutation, which affects 75,000 adults and children worldwide. Sufferers often die before the age of 40 as mucus from the disease damages their lungs, risking infection.

Cystic fibrosis sufferer
Research at Queen’s University Belfast led to the development of a transformative new drug, Ivacaftor, which has brought outstanding improvements in survival and quality of life for people with the
disease.

The breakthrough was built on a series of 25 clinical trials over 10 years which have made the university the leading UK centre for CF research. The new drug was trialled from 2009-2012 as the first approved therapy that corrects the basic defect in CF; as such it represents a completely new approach to treating people with the condition.

The Queen’s-led team of scientists found patients receiving the new treatment had significant improvement in their lung function, leading to a reduced need for hospitalisation, as well as a reduction in disease flare-ups.

Drug sales reached US$113 million in the first nine months of 2012, and Ivacaftor is now licensed and funded in the USA, UK, Ireland, France and Germany. The new drug has a very significant impact for people with CF, demonstrating real future hope that a wide range of mutations may be treatable.

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