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Modeling long-term health outcomes of patients with cystic fibrosis homozygous for F508del-CFTR treated with lumacaftor/ivacaftor

BACKGROUND: Lumacaftor/ivacaftor combination therapy is efficacious and generally safe for patients with cystic fibrosis (CF) homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. However, long-term survival benefits of lumacaftor/ivacaftor (LUM/IVA) cannot yet be quanti...

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Detalles Bibliográficos
Autores principales:	Rubin, Jaime L., O’Callaghan, Lasair, Pelligra, Christopher, Konstan, Michael W., Ward, Alexandra, Ishak, Jack K., Chandler, Conor, Liou, Theodore G.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	SAGE Publications 2019
Materias:	Original Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6366006/ https://www.ncbi.nlm.nih.gov/pubmed/30803355 http://dx.doi.org/10.1177/1753466618820186

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