Cystic fibrosis (CF) is a life-limiting genetic disorder caused by mutations in the CFTR gene. In recent years, CFTR modulators have transformed the lives of many people with CF, treating the underlying defect in the CFTR protein rather than only the symptoms of the disease.
This progress is very personal to us. Our first daughter, Moira, has cystic fibrosis, and we are incredibly grateful that she is able to benefit from these remarkable therapies. We have seen firsthand how advances in research can fundamentally change the outlook for people living with CF and their families.
But this progress has not yet reached everyone. Around 10% of people with CF cannot benefit from currently available modulator therapies, often because they have rare mutations for which these drugs do not work. For these patients and their families, there is still an urgent need for new treatments.
If you would like to help support the research needed to make effective therapies available to everyone with cystic fibrosis, please consider donating here:
Thank you!