Cystic Fibrosis (CF) is a rare, life-shortening, genetic disease affecting more than 10,000 people in the UK. It causes thick, sticky mucus to build up in the lungs, digestive system and other organs causing a wide range of challenging symptoms.
The NIHR Manchester CRF at Wythenshawe Hospital has played a key role in clinical trials for some of the most recent and powerful triple combination therapies for CF for more than 10 years and we remain at the forefront of CF therapies.
We were the lead site for the landmark triple therapy trials that led to licensing of the combination therapy Kaftrio in 2020. This treatment is now available for more than 90% of CF patients in the UK aged over 2 years.
This has had life-changing impacts on the health of CF patients, with significant improvement in lung function and reductions in hospital admissions, oxygen support and patients requiring lung transplants.
Why is further research important?
We completed early and later phase studies of the next-generation, once-daily triple therapy and are the lead site for an inhaled genetic treatment being trialled for CF patients not eligible for correcting therapies.