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Effects of lumacaftor—ivacaftor therapy on cystic fibrosis transmembrane conductance regulator function in F508del homozygous patients with cystic fibrosis aged 2–11 years

Rationale: Lumacaftor/ivacaftor was approved for the treatment of patients with cystic fibrosis who are homozygous for F508del aged 2 years and older following positive results from phase three trials. However, the improvement in CFTR function associated with lumacaftor/ivacaftor has only been studi...

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Detalles Bibliográficos
Autores principales: Berges, Julian, Graeber, Simon Y., Hämmerling, Susanne, Yu, Yin, Krümpelmann, Arne, Stahl, Mirjam, Hirtz, Stephanie, Scheuermann, Heike, Mall, Marcus A., Sommerburg, Olaf
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10266342/
https://www.ncbi.nlm.nih.gov/pubmed/37324488
http://dx.doi.org/10.3389/fphar.2023.1188051