In a landmark development, the first cystic fibrosis drug – Orkambi (lumacaftor 200 mg/ivacaftor 125 mg) – capable of treating the cause of the disease in people who have two copies of…
Tag: Cystic Fibrosis
Cystic fibrosis gene therapy looks promising
A major UK trial of a gene therapy for cystic fibrosis has shown promising results with those receiving the treatment showing significant, if modest, benefit in lung functions. Carried out at Royal…
New treatment shows promise for cystic fibrosis
A new treatment involving a combination of lumacaftor-ivacaftor is showing promise against the most common genetic cause of cystic fibrosis as the therapy improves lung function and lowers the rate of pulmonary…