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

• Autores principales: Julian Berges (Autor), Simon Y. Graeber (Autor), Susanne Hämmerling (Autor), Yin Yu (Autor), Arne Krümpelmann (Autor), Mirjam Stahl (Autor), Stephanie Hirtz (Autor), Heike Scheuermann (Autor), Marcus A. Mall (Autor), Olaf Sommerburg (Autor)
• Formato: Libro
• Publicado: Frontiers Media S.A., 2023-05-01T00:00:00Z.
• Materias: article
• Acceso en línea: Connect to this object online.

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...