Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

BACKGROUND: Cystic fibrosis is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein, and nearly 90% of patients have at least one copy of the Phe508del CFTR mutation. In a phase 2 trial involving patients who were heterozygous for the Phe508...

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Gepubliceerd in:N Engl J Med
Hoofdauteurs: Middleton, P.G., Mall, M.A., Dřevínek, P., Lands, L.C., McKone, E.F., Polineni, D., Ramsey, B.W., Taylor-Cousar, J.L., Tullis, E., Vermeulen, F., Marigowda, G., McKee, C.M., Moskowitz, S.M., Nair, N., Savage, J., Simard, C., Tian, S., Waltz, D., Xuan, F., Rowe, S.M., Jain, R.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2019
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7282384/
https://ncbi.nlm.nih.gov/pubmed/31697873
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1908639
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