Cystic Fibrosis

Cystic Fibrosis Breakthrough | Our Research Shapes Worlds

Cystic Fibrosis

Researchers from Queen’s University Belfast have transformed the lives of thousands of people with Cystic Fibrosis by leading on the clinical development of treatments that address the underlying genetic disorder.

Cystic Fibrosis is a progressive, life-limiting genetic disease that causes severe respiratory and digestive problems as well as other complications such as infections and diabetes.

There are over 80,000 people living with Cystic Fibrosis globally, including 10,500 in the UK accounting for 9,500 hospital admissions and over 100,000 bed days per year.

The condition is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene which is responsible for the regulation of salt and water levels in the body. The mutations can lead to the build-up of thick mucus in the lungs, digestive tract and other parts of the body causing persistent chest infections, resulting in lung damage and an early death.

Queen’s University’s Cystic Fibrosis research team is recognised as world leading, having worked for over 12 years supporting the development of drugs that improve the function of CTFR.

Prior to their work, treatments for Cystic Fibrosis had been focused on symptom control. However, during the last decade, Queen’s University Belfast has been at the forefront of major advancements in drugs targeting the underlying genetic deficit.

 

This work included the development of clinical trial protocols, and inclusion of key outcome measures such as; lung function (FEV1), pulmonary exacerbation rate, and Quality of Life (QoL) tools for use in clinical trials of new therapeutics.

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