There are four CFTR modulators for people with certain CFTR mutations: Trikafta® (elexacaftor/tezacaftor/ivacaftor); Symdeko® (tezacaftor/ivacaftor); Orkambi® ( ... |
CFTR modulators for the treatment of cystic fibrosis are a growing area that is quickly changing. Ivacaftor, the first CFTR potentiator to receive FDA approval, ... |
Defects in a single gene lead to the defective proteins that cause cystic fibrosis, making the disease an ideal candidate for mutation-targeted therapy. |
Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are a class of drugs that act by improving production, intracellular processing, and/or ... |
Highlights · CFTR Modulators target the underlying protein defects in CF. · Elexacaftor/tezacaftor/ivacaftor is highly effective and widely applicable. |
There are two main types of CFTR modulators: potentiators and correctors. Potentiators are CFTR modulators that hold the gate to the CFTR channel open so ... |
CFTR modulators work differently to other medications for CF. They work by correcting the malfunctioning protein that causes CF. |
Treatment of cystic fibrosis airway cells with CFTR modulators reverses aberrant mucus properties via hydration | European Respiratory Society. |
Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies work to correct the malfunctioning protein made by the CFTR gene. |
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