CFTR Potentiator VX-770 Ivacaftor CAS: 873054-44-5: A Revolutionary Treatment for Cystic Fibrosis
Introduction:
Cystic fibrosis (CF) is a genetic disorder that affects the respiratory, digestive, and reproductive systems. It is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which leads to the production of a malfunctioning protein that disrupts the balance of salt and water in various organs in the body. This results in the buildup of thick, sticky mucus that clogs airways, obstructs the pancreas, and impairs other vital functions in the body. CF is a chronic condition that has no cure, but there are treatments available that can alleviate symptoms and improve quality of life for patients.
One such treatment is the CFTR potentiator VX-770 Ivacaftor, also known by its chemical name N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide. Its molecular formula is C24H28N2O3, and its formula weight is 392.49 g/mol. The CAS number for VX-770 Ivacaftor is 873054-44-5.
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Health Benefits of this Product:
VX-770 Ivacaftor is a type of CFTR modulator, which means it targets the underlying cause of CF by correcting the defective protein produced by the CFTR gene. Specifically, VX-770 Ivacaftor is a potentiator, which means it enhances the activity of the CFTR protein by making it more responsive to signals that activate it. This allows chloride ions to move more freely across cell membranes, which helps restore the balance of salt and water in affected organs.
In clinical trials, VX-770 Ivacaftor has been shown to improve lung function, reduce exacerbations, and improve overall health outcomes in CF patients with specific gating mutations in their CFTR genes. These mutations affect how the CFTR protein opens and closes in response to signals, which impairs its ability to transport chloride ions across cell membranes. VX-770 Ivacaftor works by keeping the CFTR channel open for longer periods, which allows more chloride ions to pass through, thereby improving the function of affected organs.
Potential Effects:
VX-770 Ivacaftor has the potential to improve lung function, reduce exacerbations, and improve overall health outcomes in CF patients with specific gating mutations in their CFTR genes. In addition, it may help improve pancreatic function, reduce gastrointestinal symptoms, and increase weight gain in some patients. However, the effects of VX-770 Ivacaftor vary depending on the specific mutation present in the patient's CFTR gene, as well as other factors such as age, disease severity, and overall health status.
Product Mechanism:
VX-770 Ivacaftor works by binding to a specific site on the CFTR protein and enhancing its activity. Specifically, it binds to the "potentiation" site on the protein, which regulates its opening and closing behavior in response to signals. By keeping the channel open for longer periods, VX-770 Ivacaftor allows more chloride ions to pass through, thereby restoring the balance of salt and water in affected organs.
Safety:
VX-770 Ivacaftor is generally safe and well-tolerated in CF patients with specific gating mutations in their CFTR genes. However, like all medications, it may cause side effects in some patients. Common side effects include headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, and elevated liver enzymes. Serious adverse events are rare but may include liver toxicity, allergic reactions, and pulmonary exacerbations. Patients should be monitored closely for any signs of adverse reactions while taking VX-770 Ivacaftor.
Dosing Information:
The recommended dose of VX-770 Ivacaftor varies depending on the patient's age and weight, as well as the specific gating mutation present in their CFTR gene. In general, the approved dosages range from 75 mg twice daily to 150 mg twice daily. The medication is administered orally in tablet form and should be taken with fat-containing food to enhance absorption.
Conclusion:
VX-770 Ivacaftor is a revolutionary treatment for CF patients with specific gating mutations in their CFTR genes. It works by enhancing the activity of the CFTR protein and restoring the balance of salt and water in affected organs
