A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR

RATIONALE: Previous phase 3 trials showed that treatment with lumacaftor/ivacaftor was safe and efficacious in people aged ⩾2 years with cystic fibrosis (CF) homozygous for the F508del mutation in CFTR (CF transmembrane conductance regulator) (F/F genotype). OBJECTIVES: To assess the safety, pharmac...

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Detalles Bibliográficos
Autores principales: Rayment, Jonathan H., Asfour, Fadi, Rosenfeld, Margaret, Higgins, Mark, Liu, Lingyun, Mascia, Molly, Paz-Diaz, Hildegarde, Tian, Simon, Zahigian, Rachel, McColley, Susanna A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Thoracic Society 2022
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9746849/
https://www.ncbi.nlm.nih.gov/pubmed/35771568
http://dx.doi.org/10.1164/rccm.202204-0734OC

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9746849/
https://www.ncbi.nlm.nih.gov/pubmed/35771568
http://dx.doi.org/10.1164/rccm.202204-0734OC

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