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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 |
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| Hoofdauteurs: | , , , , , , , , , , , , , , , , , , , , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
2019
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| Onderwerpen: | |
| 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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