Lumacaftor
Lumacaftor
Lumacaftor (pronounced loo-muh-KAF-tor) is a medication used in combination with Ivacaftor for the treatment of Cystic Fibrosis in patients who have a specific genetic mutation.
Etymology
The name "Lumacaftor" is derived from the Latin word "luma," meaning light, and "caftor," a term used in pharmaceutical naming to denote a drug's function. In this case, "caftor" signifies the drug's ability to facilitate the proper function of the CFTR protein, which is often impaired in patients with Cystic Fibrosis.
Usage
Lumacaftor is used in combination with Ivacaftor, another medication, to treat Cystic Fibrosis in patients who have a specific genetic mutation known as the F508del mutation. This combination is sold under the brand name Orkambi.
Mechanism of Action
Lumacaftor works by correcting the folding and trafficking of the defective CFTR protein, allowing it to reach the cell surface. Once at the cell surface, the CFTR protein can function properly, helping to improve lung function and reduce symptoms of Cystic Fibrosis.
Related Terms
- Cystic Fibrosis: A genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine.
- Ivacaftor: A medication used in combination with Lumacaftor to treat Cystic Fibrosis.
- Orkambi: The brand name for the combination of Lumacaftor and Ivacaftor.
- F508del mutation: A specific genetic mutation that causes Cystic Fibrosis.
External links
- Medical encyclopedia article on Lumacaftor
- Wikipedia's article - Lumacaftor
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