GeneticsLiving With CFResearchTreatments

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 CFTR gating mutation. We aimed to assess the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in children aged 2–5 years.

Read more…

Previous post

Genome editing with CRISPR-Cas9

Next post

YOUR DATA! Your Questions on Self Management