Ad
related to: cystic fibrosis prevention
Search results
Results From The WOW.Com Content Network
Cystic fibrosis (CF) is a progressive genetic disease that adversely affects a person's lungs, pancreas and other vital organs, says Dr. Michael Boyle, president and CEO of the Cystic Fibrosis ...
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.
The combination is indicated for the treatment of cystic fibrosis in people aged six years of age and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. [1] [4]
Of the approximately 70,000 cases of cystic fibrosis worldwide, 4% (~3,000) are due to a mutation called G551D. [50] [51] The safety and efficacy of ivacaftor for the treatment of cystic fibrosis in patients with this mutation was examined in two clinical trials. [citation needed]
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]
Differentiation of DIOS from constipation is generally performed by a unit specializing in the treatment of cystic fibrosis. Adequate hydration and an aggressive regimen of laxatives are essential for treatment and prevention of DIOS. Osmotic laxatives such as polyethylene glycol are preferred. [1]
The Cystic Fibrosis Foundation has been a pioneer of cystic fibrosis treatment, having played a major role in the development and use of five FDA-approved therapies, including ivacaftor (Kalydeco). [7] The latest FDA approved drug is Trikafta, which was passed by the FDA in 54 days and is able to help up to 90% of CF patients. [8]
Tezacaftor is a medication used for the treatment of cystic fibrosis. [1] [2] It is available in fixed-dose combination medications.[3] [4] [5]The combination of tezacaftor with ivacaftor (brand name Symdeko) was approved for medical use in the United States in February 2018, [6] [1] and in Canada in June 2018. [7]