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Profiling the response to lumacaftor‐ivacaftor in children with cystic between fibrosis and new insight from a French‐Italian real‐life cohort

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Clinical trials for CFTR modulators consider mean changes of clinical status at the cohort level, and thus fail to assess the heterogeneity of the response. We aimed to study the… Click to show full abstract

Clinical trials for CFTR modulators consider mean changes of clinical status at the cohort level, and thus fail to assess the heterogeneity of the response. We aimed to study the different response profiles to lumacaftor‐ivacaftor according to age in children with cystic fibrosis (CF).

Keywords: response; lumacaftor ivacaftor; cohort; children cystic; cystic fibrosis

Journal Title: Pediatric Pulmonology
Year Published: 2022

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