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Ivacaftor in Infants Aged 4 to <12 Months with Cystic Fibrosis and a Gating Mutation. Results of a Two-Part Phase 3 Clinical Trial

Rationale: We previously reported that ivacaftor was safe and well tolerated in cohorts aged 12 to <24 months with cystic fibrosis and gating mutations in the ARRIVAL study; here, we report results for cohorts aged 4 to <12 months. Objectives: To evaluate the safety, pharmacokinetics, and phar...

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Detalles Bibliográficos
Autores principales: Davies, Jane C., Wainwright, Claire E., Sawicki, Gregory S., Higgins, Mark N., Campbell, Daniel, Harris, Christopher, Panorchan, Paul, Haseltine, Eric, Tian, Simon
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Thoracic Society 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7924576/
https://www.ncbi.nlm.nih.gov/pubmed/33023304
http://dx.doi.org/10.1164/rccm.202008-3177OC