Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial

Rationale: The combination of lumacaftor (LUM) and ivacaftor (IVA) is an approved CFTR (cystic fibrosis [CF] transmembrane conductance regulator) modulator treatment for homozygous F508del patients with CF. Objectives: To evaluate the effectiveness of LUM/IVA in children (6 yr or more) and adults (m...

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Vydáno v:Ann Am Thorac Soc
Hlavní autoři: Sagel, Scott D., Khan, Umer, Heltshe, Sonya L., Clancy, John P., Borowitz, Drucy, Gelfond, Daniel, Donaldson, Scott H., Moran, Antoinette, Ratjen, Felix, VanDalfsen, Jill M.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Thoracic Society 2021
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Original Research
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7780982/
https://ncbi.nlm.nih.gov/pubmed/32644818
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1513/AnnalsATS.202002-144OC
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