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Trial results of Ivacaftor in children ages 2-5

Results are out from a two part study involving children ages 2-5 with at least one gating mutation trying Ivacaftor. From the Lancet Respiratory Journal, 20 January 2016 Ivacaftor has been shown to be a safe, effective treatment for cystic fibrosis in patients aged 6 years or older with a …

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Living With CFNews & EventsTreatments

New cystic fibrosis drug has high potential, high cost

Written by Tracey Walker Source: Managed Healthcare Executive FDA’s approval of Vertex Pharmaceutical’s groundbreaking lumacaftor/ivacaftor (Orkambi) for cystic fibrosis (CF) is the first drug to treat the underlying cause of CF in people ages 12 and older with two copies of the F508del mutation. CF is an inherited chronic disease that affects …

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Living With CFResearchRespiratory DiseaseTreatments

Concert Pharma’s Modified Version of Cystic Fibrosis Therapy Ivacaftor Outperforms Kalydeco in Early Testing

Written by Patricia Inacio, PhD Source: Cystic Fibrosis News Today Concert Pharma recently announced successful results from a Phase 1 single ascending dose clinical trial for their lead investigational drug CTP-656 as a therapy for cystic fibrosis. Cystic fibrosis is a life-threatening disease characterized by a progressive lung function decline and is caused …

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