How does Kalydeco work?
Kalydeco specifically targets the underlying cause of cystic fibrosis (CF) and is approved for adults, children, and infants aged 4 months and older with CF with one or more specific mutations in their CFTR gene that is responsive to Kalydeco. It allows the defective CFTR protein to work better and increases salt and water movement within the airways. This thins the mucus and makes it easier to cough out.
Research demonstrates that Kalydeco markedly improves lung function, reduces salt levels in sweat, and helps people with CF gain weight. It is too early to say whether it will cure people with CF.
Read next
What is an orphan drug?
Orphan drugs are medicines developed to help treat, prevent or diagnose rare “orphan” diseases, which are conditions that each affect fewer than 200,000 people in the U.S. Continue reading
What is Fluimucil used for?
Fluimucil is a brand name for acetylcysteine sachets and tablets which may be used to clear phlegm and liquefy thick and viscous mucus secretions caused by respiratory conditions such as acute bronchitis, chronic bronchitis, cystic fibrosis, pneumonia, pulmonary emphysema, or bronchiectasis. It is a type of mucolytic. Fluimucil is available over the counter (without a prescription) in most countries that have it. Continue reading
What is Trikafta?
Trikafta is a triple combination therapy that contains elexacaftor, ivacaftor, and tezacaftor, which is approved for adults and children 2 years of age and older with at least one F508del mutation in the CFTR gene, or a mutation in the CFTR gene that is responsive to Trikafta based on in vitro data. Continue reading
Related medical questions
- What is the difference between Symdeko and Orkambi?
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- How does Symdeko work?
- How do you take Orkambi?
- How do the warnings differ between Symdeko and Orkambi?
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Drug information
Related support groups
- Ivacaftor (4 questions, 3 members)
- Kalydeco (3 questions, 5 members)
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