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Cystic Fibrosis with Ileus News

Cystic Fibrosis Drug Seems OK for Preschoolers: Study

Posted 25 Jan 2016 by Drugs.com

MONDAY, Jan. 25, 2016 – The cystic fibrosis drug ivacaftor appears safe and effective for young children, a drug company-funded study suggests. "This was a small trial, but we are thrilled to see these results," said study leader Jane Davies, from the National Heart and Lung Institute at Imperial College London in England. "Ivacaftor is a potential new treatment to offer children aged 2 years and older with cystic fibrosis and a [specific gene mutation linked to the disease]. This novel therapy could substantially impact these children's lives, potentially opening the way to even greater progress in years to come." Cystic fibrosis is a life-threatening genetic disease that destroys the lungs and digestive system. More than 70,000 people worldwide have cystic fibrosis, the researchers said. One expert explained that ivacaftor has been considered a big advance in the treatment of the ... Read more

Related support groups: Cystic Fibrosis, Orkambi, Ivacaftor, Cystic Fibrosis with Ileus, Kalydeco, Ivacaftor/lumacaftor

Gene Therapy for Cystic Fibrosis Shows Some Promise in Study

Posted 2 Jul 2015 by Drugs.com

THURSDAY, July 2, 2015 – Gene therapy for cystic fibrosis has for the first time shown slight but significant benefit on lung function, new British research reveals. In a randomized trial, patients inhaled molecules of DNA that aimed to replace the defective gene responsible for cystic fibrosis with a healthy, working copy of the gene in the lungs. "Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group, and there were no safety concerns," study senior author Eric Alton of the National Heart and Lung Institute at Imperial College London, said in a news release fromThe Lancet Respiratory Medicine, which published the study. Even though the effect was "inconsistent, with some patients responding better than others, the results are encouraging," Alton said. Cystic fibrosis is a rare, inherited respiratory ... Read more

Related support groups: Cystic Fibrosis, Cystic Fibrosis with Ileus

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