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Lumacaftor-ivacaftor in the treatment of cystic fibrosis: design, development and place in therapy

Lumacaftor-ivacaftor is a combination of two small molecule therapies targeting the basic defect in cystic fibrosis (CF) at a cellular level. It is a precision medicine and its effects are specific to individuals with two copies of the p.Phe508del gene mutation. The drug combination works by restori...

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Detalles Bibliográficos
Autor principal: Connett, GJ
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Dove 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6650604/
https://www.ncbi.nlm.nih.gov/pubmed/31409974
http://dx.doi.org/10.2147/DDDT.S153719

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