CFTRmodulaattorit

CFTRmodulaattorit are a class of drugs used to treat cystic fibrosis (CF), a genetic disorder that affects the lungs and other organs. These drugs work by modulating the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is defective in people with CF. CFTRmodulaattorit are typically used in combination with other CF treatments, such as antibiotics and inhaled medications.

There are several types of CFTRmodulaattorit, including potentiators and correctors. Potentiators, such as ivacaftor, work by increasing the activity of the CFTR protein, allowing it to function more effectively. Correctors, such as tezacaftor and givosiran, work by correcting the underlying genetic mutation in the CFTR gene, allowing the protein to be produced and function properly.

CFTRmodulaattorit have been shown to improve lung function and reduce the frequency of respiratory infections in people with CF. However, they are not a cure for the disease and must be used in combination with other treatments. Additionally, CFTRmodulaattorit can have side effects, such as diarrhea and abdominal pain, and may not be effective for everyone with CF.

CFTRmodulaattorit were first approved by the FDA in 2012, and since then, several new drugs in this class have been developed and approved. The availability of CFTRmodulaattorit has significantly improved the quality of life for people with CF and has opened up new possibilities for future treatments.