Cystic Fibrosis Channel
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What Is Kalydeco?

If you have cystic fibrosis, your healthcare provider may recommend treatment with a medication called Kalydeco™ (ivacaftor). But what is this medication and what can you expect during treatment with it?
While it is an exciting breakthrough for cystic fibrosis treatment, Kalydeco is only effective for use in people who have certain gene mutations, including G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. It is not effective in people with the most common cystic fibrosis mutation (F508del mutation).
Clinical studies have shown that this medication can improve lung function, reduce symptoms, help with weight gain, and reduce the chance of pulmonary exacerbations. Kalydeco is approved for use in adults and children as young as six years old. It comes as a tablet that is taken once daily, twice daily, or twice a week.
Although many people tolerate this prescription drug well, side effects can occur and may include headaches and throat pain.
(To learn more, click Kalydeco. This full-length article contains information on various topics, including how this drug works, dosing instructions, and safety precautions to review before beginning treatment.)
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