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Please note: While some information will still be current in a year, other information may already be out of date in three months time. If you are in any doubt, please feel free to ask.
CF 2013
- Question
- Essence of question: is there any information of new medicines improving the underlying defect in CF?
- Answer
- Dear questioner,
You ask about information of new medicines aimed at improving the underlying defect in CF. This research has progressed well in the last years.
The first medicine that improves the underlying defect in CF is on the market. This drug, ivacaftor (Kaleydeco®) ensures a better function of the CFTR chloride channel in patients who carry the G551D mutation. Patients with this mutation treated with this drug had better lung function, better weight, felt better and had less exacerbations of lung infection. Unfortunately, this mutation is only present in about 4% of CF patients worldwide. It is currently being tested in patients who carry other class 3 mutations (about 1% of patients with CF).
Best regards,
Prof. S. Dumcius
- 27.05.2013
- Please find more information under:
ecorn-cf.eu/index.php?id=65&L=8&tx_expertadvice_pi1%5Bshowitem%5D=2423&tx_expertadvice_pi1%5Bsearch%5D=VX%20809
ecorn-cf.eu/index.php?id=65&L=8&tx_expertadvice_pi1%5Bshowitem%5D=2199&tx_expertadvice_pi1%5Bsearch%5D=VX%20809
ecorn-cf.eu/index.php?id=65&L=8&tx_expertadvice_pi1%5Bshowitem%5D=2161&tx_expertadvice_pi1%5Bsearch%5D=VX%20809
D. d'Alquen
