Gayle Pledger is the co-founder of campaign group Vertex Save Us and parent to a daughter with CF. In this blog, she writes about how she became involved with the campaign for global access to Kaftrio and what it means to her, and shares an update on her fight to get cystic fibrosis treatments included on the World Health Organization’s (WHO) Essential Medicines List.
The National Institute for Health and Care Excellence (NICE) have today (15 July) given approval for the once-a-day triple therapy, Alyftrek (vanza triple). At the same time, NHS England have confirmed an extension to eligibility for Kaftrio and Alyftrek - giving access to more people with CF. Announcements are expected to follow in Scotland, Northern Ireland and Wales.
Cystic Fibrosis Trust responds to a report on medicines shortages in England, and calls on you to share your experiences accessing Pancreatic Enzyme Replacement Therapy (PERT) in 2025.
Cracking the CF Code is our new resource for children with CF. It was completely written by young people in our Youth Advisory Group (YAG), who used their knowledge and experiences to explain tricky medical words and concepts to 6–9-year olds. We go behind the scenes with Ellie and Bahar, who work in the Trust’s youth team, to find out how the idea was brought to life.
This month, our dedicated supporters joined us in raising awareness of cystic fibrosis for CF Week, and continue to take on fundraising challenges for the Trust.
Ataluren is a novel investigational drug which works by helping the body to ignore the nonsense mutation and make a complete and functional CFTR protein. So far the results from clinical trials in ataluren have shown that the drug appears to be safe and well tolerated. There is currently an ongoing clinical trial (PTC124-GD-021-CF) which assesses the safety and effectiveness of ataluren in 208 patients worldwide with nmCF who are 6 years or older. After 48 weeks of treatment with ataluren or placebo, patients who successfully complete this study will have the chance to go onto this extension study (PTC124-GD-021e-CF)...
Over the next five years, our Translational Innovation Hub Network for lung health and infection in CF will address challenges around finding new lung infection treatment options, detecting and treating flare-ups, and developing new ways to diagnose infections.
