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Following the listing of the combination on the Pharmaceutical Benefits Scheme in 2022, the cost for people aged twelve years of age or older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene is $30.00 per month, or $7.30 for concession card holders. [48]
Cystic fibrosis (also known as CF or mucoviscidosis) is an autosomal recessive genetic disorder affecting most critically the lungs, and also the pancreas, liver, and intestine.
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. [ 5 ][ 6 ] It is also included in combination medications, lumacaftor/ivacaftor, tezacaftor/ivacaftor ...
(Reuters) -Vertex Pharmaceuticals' experimental cystic-fibrosis treatment met all of its main goals in a late-stage study when tested in patients aged 12 years and older, the company said on Monday.
Lumacaftor/ivacaftor. Lumacaftor/ivacaftor, sold under the brand name Orkambi among others, is a combination of lumacaftor and ivacaftor used to treat people with cystic fibrosis who have two copies of the F508del mutation. [6] It is unclear if it is useful in cystic fibrosis due to other causes. [6] It is taken by mouth.
August 1, 2024 at 4:08 PM. (Reuters) - Vertex Pharmaceuticals raised its annual revenue forecast on Thursday, betting on robust demand for its cystic fibrosis (CF) treatments. The drugmaker now ...
The Cystic Fibrosis Foundation (CFF) is a 501 (c) (3) non-profit organization in the United States established to provide the means to cure cystic fibrosis (CF) and ensure that those living with CF live long and productive lives. The Foundation provides information about cystic fibrosis and finances CF research that aims to improve the quality ...
In 2012 ivacaftor was designated as an orphan drug, identifying cystic fibrosis as affecting fewer than 200,000 people in the United States.On 31 January 2012, Vertex gained FDA approval [22] of the first drug, Kalydeco, [23] to treat the underlying cause of cystic fibrosis rather than the symptoms, in patients 6 years or older who have the G551D gene mutation.