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Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
BACKGROUND: Cystic fibrosis is a life-limiting disease that is caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is the most common CFTR mutation. METHODS: We conducted two phase 3, randomized, double-blind, placebo-controlled stu...
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| Argitaratua izan da: | N Engl J Med |
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| Egile Nagusiak: | , , , , , , , , , , , , , , , , , , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
2015
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| Gaiak: | |
| Sarrera elektronikoa: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4764353/ https://ncbi.nlm.nih.gov/pubmed/25981758 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1056/NEJMoa1409547 |
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