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(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.
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]
As a treatment administered only once, it carries an enormous $2.2 million list price. ... a new once-daily pill for patients with cystic fibrosis caused by at least one of 303 possible mutations ...
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]
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]
A child who received a life-changing cystic fibrosis drug has told how she felt better within hours. Kate Farrer, seven, is one of the first young children to be given Kaftrio on the NHS.
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.
Vertex has built an empire in cystic fibrosis (CF) treatment, recently crowning its dominance with Alyftrek's approval last month. This latest CF drug adds even more firepower to a franchise ...
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