Article
Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study.
The Lancet. Respiratory medicine - 1 Jul 2018
Rosenfeld Margaret, Wainwright Claire E, Higgins Mark, Wang Linda T, McKee Charlotte, Campbell Daniel, Tian Simon, Schneider Jennifer, Cunningham Steve, Davies Jane C
Abstract excerpt
BACKGROUND: Ivacaftor is generally safe and effective in patients aged 2 years and older who have cystic fibrosis and specific CFTR mutations. We assessed its use in children aged 12 to <24 months. METHODS: The ARRIVAL study is a phase 3, single-arm, two-part, multicentre study. Eligible children were aged 12 to <24 months at enrolment and had a confirmed diagnosis of cystic fibrosis and a CFTR gating mutation on...
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