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Trikafta
Elexacaftor/tezacaftor/ivacaftor, sold under the brand names Trikafta (US) and Kaftrio (EU), is a fixed-dose combination medication used to treat cystic fibrosis. Elexacaftor/tezacaftor/ivacaftor is composed of a combination of ivacaftor, a chloride channel opener, and elexacaftor and tezacaftor, CFTR modulators. It is approved for use in the United States for people aged six years and older who have cystic fibrosis with a F508del mutation or other mutations in the CFTR gene. It is also approved for use in Canada, the European Union and Australia. Medical uses The combination is indicated for the treatment of people aged six years and older who have cystic fibrosis with a F508del mutation or other mutations in the CFTR gene. Side effects The most common side effects affecting more than 5% of patients are headache, upper respiratory tract infection, abdominal pain, diarrhea, rash, alanine aminotransferase increase, nasal congestion, blood creatine phosphokinase increase, ...
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Elexacaftor
Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It is available in a single pill with ivacaftor and tezacaftor; the fixed-dose combination, elexacaftor/tezacaftor/ivacaftor (brand name ''Trikafta''), is used to treat people with cystic fibrosis who are homozygous for the f508del mutation. This combination was approved for medical use in the United States in 2019. The fixed-dose combination elexacaftor/tezacaftor/ivacaftor (Kaftrio) was approved for medical use in the European Union The European Union (EU) is a supranational political and economic union of member states that are located primarily in Europe. The union has a total area of and an estimated total population of about 447million. The EU has often been ... in August 2020, for the treatment of cystic fibrosis. References External links * Breakthrough therapy Cystic fibrosis Orphan drugs {{respiratory-system-drug-stub ...
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Cystic Fibrosis
Cystic fibrosis (CF) is a rare genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. Other signs and symptoms may include sinus infections, poor growth, fatty stool, clubbing of the fingers and toes, and infertility in most males. Different people may have different degrees of symptoms. Cystic fibrosis is inherited in an autosomal recessive manner. It is caused by the presence of mutations in both copies of the gene for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Those with a single working copy are carriers and otherwise mostly healthy. CFTR is involved in the production of sweat, digestive fluids, and mucus. When the CFTR is not functional, secretions which are usually thin instead become thick. The condition is diagnosed by a sweat test and genetic testing. Screening of infants at bi ...
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Elexacaftor
Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It is available in a single pill with ivacaftor and tezacaftor; the fixed-dose combination, elexacaftor/tezacaftor/ivacaftor (brand name ''Trikafta''), is used to treat people with cystic fibrosis who are homozygous for the f508del mutation. This combination was approved for medical use in the United States in 2019. The fixed-dose combination elexacaftor/tezacaftor/ivacaftor (Kaftrio) was approved for medical use in the European Union The European Union (EU) is a supranational political and economic union of member states that are located primarily in Europe. The union has a total area of and an estimated total population of about 447million. The EU has often been ... in August 2020, for the treatment of cystic fibrosis. References External links * Breakthrough therapy Cystic fibrosis Orphan drugs {{respiratory-system-drug-stub ...
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Elexacaftor Tezacaftor Ivacaftor Mechanism Of Action
Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It is available in a single pill with ivacaftor and tezacaftor; the fixed-dose combination, elexacaftor/tezacaftor/ivacaftor (brand name ''Trikafta''), is used to treat people with cystic fibrosis who are homozygous for the f508del mutation. This combination was approved for medical use in the United States in 2019. The fixed-dose combination elexacaftor/tezacaftor/ivacaftor (Kaftrio) was approved for medical use in the European Union The European Union (EU) is a supranational political and economic union of member states that are located primarily in Europe. The union has a total area of and an estimated total population of about 447million. The EU has often been ... in August 2020, for the treatment of cystic fibrosis. References External links * Breakthrough therapy Cystic fibrosis Orphan drugs {{respiratory-system-drug-stub ...
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Ivacaftor
Ivacaftor is a medication used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (primarily the G551D mutation), who account for 4–5% cases of cystic fibrosis. It is also included in combination medications, lumacaftor/ivacaftor, tezacaftor/ivacaftor, and elexacaftor/tezacaftor/ivacaftor which are used to treat people with cystic fibrosis. Ivacaftor was developed by Vertex Pharmaceuticals in conjunction with the Cystic Fibrosis Foundation and is the first medication that treats the underlying cause rather than the symptoms of the disease. It was approved by the U.S. Food and Drug Administration (FDA) in January 2012. It is one of the most expensive drugs, costing over per year, which has led to criticism of the high cost. The combination drug lumacaftor/ivacaftor was approved by the FDA in July 2015. Cystic fibrosis is caused by any one of several defects in the CFTR protein, which regulates flui ...
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Tezacaftor
Tezacaftor is a drug used for the treatment of cystic fibrosis (CF) in people six years and older, who have a F508del mutation, the most common type of mutation in the CFTR gene. It is sold as a fixed-dose combination with ivacaftor under the brand name Symdeko. It was approved by the U.S. FDA in 2018. The combination of elexacaftor, tezacaftor, and ivacaftor is being sold as Trikafta. In 2019, the U.S. Food and Drug Administration (FDA) approved a combination of elexacaftor, tezacaftor, and ivacaftor. Mechanism of action Tezacaftor acts as a corrector to help the folding and presentation of the CFTR protein to the cell surface, which improves its function for individuals with a F508del mutation. Clinical trials The EVOLVE and EXPAND study findings were published in 2017. EVOLVE trial The EVOLVE trial analyzed tezacaftor/ivacaftor in patients with cystic fibrosis, specifically with the homozygous for Phe508del mutation. The EVOLVE trial is a phase 3, double-blinded, mul ...
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Glyburide
Glibenclamide, also known as glyburide, is an antidiabetic medication used to treat type 2 diabetes. It is recommended that it be taken together with diet and exercise. It may be used with other antidiabetic medication. It is not recommended for use by itself in type 1 diabetes. It is taken by mouth. Common side effects include nausea and heartburn. Serious side effects may include angioedema and low blood sugar. It is generally not recommended during pregnancy but can be used during breastfeeding. It is in the sulfonylureas class of medications and works by increasing the release of insulin from the pancreas. Glibenclamide was discovered in 1969 and approved for medical use in the United States in 1984. It is available as a generic medication. In 2022, it was the 200th most commonly prescribed medication in the United States, with more than 2million prescriptions. Medical uses Glibenclamide is indicated as an adjunct to diet and exercise to improve glycemic control in ad ...
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Warfarin
Warfarin, sold under the brand name Coumadin among others, is a medication that is used as an anticoagulant (blood thinner). It is commonly used to prevent blood clots such as deep vein thrombosis and pulmonary embolism, and to prevent stroke in people who have atrial fibrillation, valvular heart disease, or artificial heart valves. Less commonly, it is used following ST-segment elevation myocardial infarction and orthopedic surgery. It is generally taken by mouth, but may also be used intravenously. The common side effect is bleeding. Less common side effects may include areas of tissue damage and purple toes syndrome. Use is not recommended during pregnancy. The effects of warfarin typically should be monitored by checking prothrombin time (INR) every one to four weeks. Many other medications and dietary factors can interact with warfarin, either increasing or decreasing its effectiveness. The effects of warfarin may be reversed with phytomenadione (vitamin ...
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Digoxin
Digoxin (better known as Digitalis), sold under the brand name Lanoxin among others, is a medication used to treat various heart conditions. Most frequently it is used for atrial fibrillation, atrial flutter, and heart failure. Digoxin is one of the oldest medications used in the field of cardiology. It works by increasing myocardial contractility, increasing stroke volume and blood pressure, reducing heart rate, and somewhat extending the time frame of the contraction. Digoxin is taken by mouth or by injection into a vein. Digoxin has a half life of approximately 36 hours given at average doses in patients with normal renal function. It is excreted mostly unchanged in the urine. Common side effects include breast enlargement with other side effects generally due to an excessive dose. These side effects may include loss of appetite, nausea, trouble seeing, confusion, and an irregular heartbeat. Greater care is required in older people and those with poor kidney function. ...
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Statin
Statins, also known as HMG-CoA reductase inhibitors, are a class of lipid-lowering medications that reduce illness and mortality in those who are at high risk of cardiovascular disease. They are the most common cholesterol-lowering drugs. Low-density lipoprotein (LDL) carriers of cholesterol play a key role in the development of atherosclerosis and coronary heart disease via the mechanisms described by the lipid hypothesis. Statins are effective in lowering LDL cholesterol and so are widely used for primary prevention in people at high risk of cardiovascular disease, as well as in secondary prevention for those who have developed cardiovascular disease. Side effects of statins include muscle pain, increased risk of diabetes mellitus, and abnormal blood levels of liver enzymes. Additionally, they have rare but severe adverse effects, particularly muscle damage. They inhibit the enzyme HMG-CoA reductase which plays a central role in the production of cholesterol. High ...
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Complex Of Ivacaftor Bound To CFTR
Complex commonly refers to: * Complexity, the behaviour of a system whose components interact in multiple ways so possible interactions are difficult to describe ** Complex system, a system composed of many components which may interact with each other * Complex (psychology), a core pattern of emotions etc. in the personal unconscious organized around a common theme such as power or status Complex may also refer to: Arts, entertainment and media * Complex (English band), formed in 1968, and their 1971 album ''Complex'' * Complex (band), a Japanese rock band * ''Complex'' (album), by Montaigne, 2019, and its title track * ''Complex'' (EP), by Rifle Sport, 1985 * "Complex" (song), by Gary Numan, 1979 * Complex Networks, publisher of magazine ''Complex'', now online Biology * Protein–ligand complex, a complex of a protein bound with a ligand * Exosome complex, a multi-protein intracellular complex * Protein complex, a group of two or more associated polypeptide chains * Specie ...
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