To improve medical care, it is important to collect information about the quality of life of people who receive medicines and also that of their parents. Vertex Pharmaceuticals Inc., the company who developed Orkambi and Symkevi for the treatment of Cystic Fibrosis, is conducting this observational study at hospitals in the UK to collect additional information on the quality of life of 6-11 year olds receiving these medicines and that of their parents. For those who are eligible to participate, after signing the data consent form, participants’ medical records relating to the history of their CF disease, including current and...
The main aim of the study is to assess safety and effectiveness of the CFTR modulator combination therapy Galicaftor/Navocaftor/ABBV-119 in adult participants with CF who are homozygous or heterozygous for the F508del mutation in each arm. Galicaftor/Navocaftor/ABBV-119 combination therapy is an investigational therapy for the treatment of CF. Galicaftor (CFTR corrector), Navocaftor (CFTR potentiator) and ABBV-119 (CFTR corrector) are CFTR modulators which aim to help the faulty CFTR protein to function properly. Study doctors place participants into 1 of 4 treatment groups, called treatment arms. Each group receives a different treatment regime. Around 90 adult participants with a diagnosis of...
With the start of Ramadan just under a month away, we’re sharing the story of Kareem, who has CF. Kareem and his sister Yasmin, who are both part of our Youth Advisory Group (YAG), recently reviewed our information for Muslims with CF who are making decisions about fasting during the month of Ramadan.
For us to better understand the true cost of CF, and how we can better support the community, we've put together a short anonymous survey to get a true representation of what our community faces day-to-day.
