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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

INTRODUCTION: 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)....

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Detalles Bibliográficos
Autores principales: Cornet, Matthieu, Robin, Geneviève, Ciciriello, Fabiana, Bihouee, Tiphaine, Marguet, Christophe, Roy, Valérie, Lebourgeois, Muriel, Chedevergne, Frédérique, Bonnel, Anne Sophie, Kelly, Mairead, Reix, Philippe, Lucidi, Vincenzina, Stoven, Véronique, Sermet‐Gaudelus, Isabelle
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9826158/
https://www.ncbi.nlm.nih.gov/pubmed/35996214
http://dx.doi.org/10.1002/ppul.26123