Transforming the lives of people with Cystic Fibrosis

Published by: Queens University- Belfast

A Queen’s University research team 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.

Summary of the impact

The Queen’s University 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 have 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.

Extensive clinical trial experience coupled with the Clinical Trial Network infrastructure established by Queen’s and the Belfast Health and Social Care Trust, resulted in Queen’s playing a pivotal role in a drug development programme working alongside Vertex Pharmaceuticals to deliver trials for single, double and triple therapies in Cystic Fibrosis.

Working with industry, clinical trial networks and contract research organisations and colleagues at other Higher Education Institutes such as Imperial College, Queen’s University has developed expertise in the delivery of clinical trials of single and multiple combination therapies. Click here to read the rest of the story.

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