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

• Автори: Julian Berges (Автор), Simon Y. Graeber (Автор), Susanne Hämmerling (Автор), Yin Yu (Автор), Arne Krümpelmann (Автор), Mirjam Stahl (Автор), Stephanie Hirtz (Автор), Heike Scheuermann (Автор), Marcus A. Mall (Автор), Olaf Sommerburg (Автор)
• Формат: Книга
• Опубліковано: Frontiers Media S.A., 2023-05-01T00:00:00Z.
• Предмети: article
• Онлайн доступ: 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...