Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator gene (CFTR) that result in diminished quantity and/or function of the CFTR anion channel. F508del-CFTR, the most common CF-causing mutation (found in ∼90% of patients), causes severe processing and trafficking...

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Detalles Bibliográficos
Autores principales: Taylor-Cousar, Jennifer L., Mall, Marcus A., Ramsey, Bonnie W., McKone, Edward F., Tullis, Elizabeth, Marigowda, Gautham, McKee, Charlotte M., Waltz, David, Moskowitz, Samuel M., Savage, Jessica, Xuan, Fengjuan, Rowe, Steven M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: European Respiratory Society 2019
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6571452/
https://www.ncbi.nlm.nih.gov/pubmed/31218221
http://dx.doi.org/10.1183/23120541.00082-2019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6571452/
https://www.ncbi.nlm.nih.gov/pubmed/31218221
http://dx.doi.org/10.1183/23120541.00082-2019

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