This study is aiming to learn more about the safety and tolerability of the combination modulator treatment made up of Elexacaftor (ELX), Tezacaftor (TEZ) and Ivacaftor (IVA) (also known by its brand name Kaftrio) in children aged 12 to less than 24 months. This treatment is already approved for use in the UK for certain groups of people with CF, but has not yet been approved for this age group. There will be two parts to the study: Part A and Part B. Children taking part in Part A will receive ELX/TEZ/IVA in the morning and IVA in the evening...
The UK CF Registry is a secure centralised database, sponsored and managed by Cystic Fibrosis Trust, that records vital data on people with cystic fibrosis (CF).
Cerys, who spent the week doing work experience with us, speaks about growing up with CF, her experience of Kaftrio, life at university and her plans for the future.
We spoke with Harry, father to nine-month-old Kenny who has cystic fibrosis, about Kenny’s life with CF, navigating CF as a new parent, and advice for other families.