A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele

Rationale: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to be efficacious and safe in patients ≥12 years of age with cystic fibrosis and at least one F508del-CFTR (cystic fibrosis transmembrane conductance regulator) allele, but it has not been evaluated in children <12 years of age....

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Detalles Bibliográficos
Autores principales: Zemanick, Edith T., Taylor-Cousar, Jennifer L., Davies, Jane, Gibson, Ronald L., Mall, Marcus A., McKone, Edward F., McNally, Paul, Ramsey, Bonnie W., Rayment, Jonathan H., Rowe, Steven M., Tullis, Elizabeth, Ahluwalia, Neil, Chu, Chenghao, Ho, Thang, Moskowitz, Samuel M., Noel, Sabrina, Tian, Simon, Waltz, David, Weinstock, Tanya G., Xuan, Fengjuan, Wainwright, Claire E., McColley, Susanna A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Thoracic Society 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8483230/
https://www.ncbi.nlm.nih.gov/pubmed/33734030
http://dx.doi.org/10.1164/rccm.202102-0509OC

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