The New England journal of medicine, 2021-08, Vol.385 (9), p.815-825
- Barry, Peter J
- Mall, Marcus A
- Álvarez, Antonio
- Colombo, Carla
- de Winter-de Groot, Karin M
- Fajac, Isabelle
- McBennett, Kimberly A
- McKone, Edward F
- Ramsey, Bonnie W
- Sutharsan, Sivagurunathan
- Taylor-Cousar, Jennifer L
- Tullis, Elizabeth
- Ahluwalia, Neil
- Jun, Lucy S
- Moskowitz, Samuel M
- Prieto-Centurion, Valentin
- Tian, Simon
- Waltz, David
- Xuan, Fengjuan
- Zhang, Yaohua
- Rowe, Steven M
- Polineni, Deepika
2021
Details
- Autor(en) / Beteiligte
- Barry, Peter J
- Mall, Marcus A
- Álvarez, Antonio
- Colombo, Carla
- de Winter-de Groot, Karin M
- Fajac, Isabelle
- McBennett, Kimberly A
- McKone, Edward F
- Ramsey, Bonnie W
- Sutharsan, Sivagurunathan
- Taylor-Cousar, Jennifer L
- Tullis, Elizabeth
- Ahluwalia, Neil
- Jun, Lucy S
- Moskowitz, Samuel M
- Prieto-Centurion, Valentin
- Tian, Simon
- Waltz, David
- Xuan, Fengjuan
- Zhang, Yaohua
- Rowe, Steven M
- Polineni, Deepika
- Titel
- Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes
- Ist Teil von
- The New England journal of medicine, 2021-08, Vol.385 (9), p.815-825
- Ort / Verlag
- United States: Massachusetts Medical Society
- Erscheinungsjahr
- 2021
- Link zum Volltext
- Quelle
- Elektronische Zeitschriftenbibliothek - Frei zugängliche E-Journals
- Beschreibungen/Notizen
- Elexacaftor–ivacaftor–tezacaftor therapy is efficacious in cystic fibrosis among patients with at least one copy of the Phe508del allele. In this trial involving patients with either a gating or residual function allele in addition to the Phe508del allele, elexacaftor–ivacaftor–tezacaftor improved lung function as compared with active control therapy.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0028-4793eISSN: 1533-4406DOI: 10.1056/NEJMoa2100665Titel-ID: cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8982185
- Format
- –
- Schlagworte
- Adolescent, Adolescent Medicine, Adult, Adverse events, Alleles, Aminophenols, Aminophenols - adverse effects, Aminophenols - therapeutic use, Benzodioxoles, Benzodioxoles - adverse effects, Benzodioxoles - therapeutic use, Child, Chloride, Chloride Channel Agonists, Chloride Channel Agonists - adverse effects, Chloride Channel Agonists - therapeutic use, Chlorides, Chlorides - analysis, Collaboration, COVID-19, Critical Care, Critical Care General, Cystic Fibrosis, Cystic Fibrosis - drug therapy, Cystic Fibrosis - genetics, Cystic Fibrosis Transmembrane Conductance Regulator, Cystic Fibrosis Transmembrane Conductance Regulator - genetics, Design, Double-Blind Method, Drug Combinations, Drug therapy, Drug therapy, Combination, Female, Gating, Genetics, Genetics General, Genotype, Genotypes, Human health and pathology, Humans, Indoles, Indoles - adverse effects, Indoles - therapeutic use, Life Sciences, Male, Membrane proteins, Mutation, Patient outcomes, Patients, Pediatrics, Pharmaceuticals, Proteins, Pulmonary, Pyrazoles, Pyrazoles - adverse effects, Pyrazoles - therapeutic use, Pyridines, Pyridines - adverse effects, Pyridines - therapeutic use, Quality of life, Quinolines, Quinolines - adverse effects, Quinolines - therapeutic use, Statistical analysis, Sweat, Sweat - chemistry, Teenagers, Testing