Long-Term Impact of Lumacaftor/Ivacaftor Treatment on Cystic Fibrosis Disease Progression in Children 2 Through 5 Years of Age Homozygous for F508del-CFTR: A Phase 2, Open-Label Clinical Trial
Ann Am Thorac Soc. 2024 Aug 22. doi: 10.1513/AnnalsATS.202402-201OC. Online ahead of print. ABSTRACT RATIONALE: Clinical trials show that lumacaftor/ivacaftor (LUM/IVA) treatment has the potential …