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MAC & Bronchiectasis | Last Active: 5 days ago | Replies (17)
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@tw508 I appreciate your information. Here is what I searched for. My mother also died of lung cancer at age of 77. My grandfather got TB when he served in the army. I will get into more research in CF and related mutation. I believe it will benefit most of the patients in MAC/BE forum too.
Trikafta (elexacaftor/tezacaftor/ivacaftor) is a revolutionary triple-combination medicine for Cystic Fibrosis (CF), targeting specific genetic mutations like F508del, and works as a CFTR modulator to help the protein function better, treating CF in patients 2+ years with responsive mutations, but requires liver monitoring due to risks of serious liver damage, with S895N being a specific but less common CF mutation often responsive to this therapy.
What Trikafta Does
Mechanism: It's a CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator that corrects the faulty CFTR protein, helping it move to the cell surface and function correctly, improving mucus clearance.
Components: Contains elexacaftor, tezacaftor (correctors), and ivacaftor (potentiator).
Responsiveness: Trikafta is indicated for patients with at least one F508del mutation or other mutations, that are responsive to the drug based on clinical data.
Key Considerations
Liver Issues: A major warning is potential serious liver damage or failure, requiring regular blood tests.
Dosage: Taken orally, usually twice daily, with fat-containing food.
Eligibility: For CF patients aged 2 and older with at least one F508del or another responsive mutation.