CF Patients With Rare Mutation May Also Respond Well to Kalydeco and Orkambi


Knowing exactly how rare mutations affect the CFTR gene in patients with cystic fibrosis (CF) helps researchers find more suitable treatments for CF patients.

Want to learn more about CFTR and CF genetics? Here are seven facts about it.

According to a recent study about the abnormalities associated with the rare P67L mutation, there are already approved drugs that can target this dysfunctional protein, improving its ability to work.

The P67L mutation affects a small number of individuals with CF, which is why not much is known about its impact on the function of the CFTR protein and the consequences to patients.

Learn more about this study here.

Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.