Nearly 300 children with cystic fibrosis and two copies of the F508del mutation are now eligible for the first time for a medicine that can treat the underlying cause of their disease.
Nearly 300 children with cystic fibrosis and two copies of the F508del mutation are now eligible for the first time for a medicine that can treat the underlying cause of their disease.
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Cystic fibrosis patients being treated with a CFTR modulator had more positive outlooks on their futures, treatment plans, and healthcare professionals.
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According to BBC UK, parents of children with cystic fibrosis have resorted to medical tourism to purchase a generic of the CF drug Orkambi, which is not available through the National Health Service in the UK.
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Orkambi appeared to improve lung function and body weight and reduce the need for intravenous antibiotic treatment in adult and pediatric cystic fibrosis patients, but 18% of patients did not tolerate the therapy.
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A pharmaceutical advisory committee in Canada has recommended that Orkambi (lumacaftor/ivacaftor) not be reimbursed for the treatment of cystic fibrosis.
Read MoreCystic fibrosis patients are fighting for access to Vertex Pharma’s $250,000/year drug Orkambi.
Read MoreVertex Pharmaceuticals Inc reports that its cystic fibrosis drug Orkambi remains on track to generate sales of at least $1 billion this year.
Read MoreThe FDA has approved Vertex Pharma’s Orkambi, the first drug directed at treating cystic fibrosis patients 12 years and older with two copies of the F508del mutation.
Read MoreVertex’s investigational drug Orkambi (lumacaftor/ivacaftor) could potentially treat 8,500 Americans with cystic fibrosis aged 12 and older with two copies of the F508del mutation.
Read MoreVertex’s pipeline combination lumacaftor-ivacaftor drug is indicated for the treatment of cystic fibrosis in patients carrying the F508del CFTR gene alteration.
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