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Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes
by
Moskowitz, Samuel M
, Polineni, Deepika
, Fajac, Isabelle
, Ramsey, Bonnie W
, Tullis, Elizabeth
, Ahluwalia, Neil
, Tian, Simon
, Barry, Peter J
, Álvarez, Antonio
, de Winter-de Groot, Karin M
, Mall, Marcus A
, Taylor-Cousar, Jennifer L
, Zhang, Yaohua
, Waltz, David
, Xuan, Fengjuan
, Rowe, Steven M
, Colombo, Carla
, Jun, Lucy S
, Sutharsan, Sivagurunathan
, McBennett, Kimberly A
, McKone, Edward F
, Prieto-Centurion, Valentin
in
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
/ Female
/ Gating
/ Genetics
/ Genetics General
/ Genotype
/ Genotypes
/ Human health and pathology
/ Humans
/ Indoles
/ Indoles - adverse effects
/ Indoles - therapeutic use
/ Life Sciences
/ Male
/ Mutation
/ 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
2021
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Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes
by
Moskowitz, Samuel M
, Polineni, Deepika
, Fajac, Isabelle
, Ramsey, Bonnie W
, Tullis, Elizabeth
, Ahluwalia, Neil
, Tian, Simon
, Barry, Peter J
, Álvarez, Antonio
, de Winter-de Groot, Karin M
, Mall, Marcus A
, Taylor-Cousar, Jennifer L
, Zhang, Yaohua
, Waltz, David
, Xuan, Fengjuan
, Rowe, Steven M
, Colombo, Carla
, Jun, Lucy S
, Sutharsan, Sivagurunathan
, McBennett, Kimberly A
, McKone, Edward F
, Prieto-Centurion, Valentin
in
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
/ Female
/ Gating
/ Genetics
/ Genetics General
/ Genotype
/ Genotypes
/ Human health and pathology
/ Humans
/ Indoles
/ Indoles - adverse effects
/ Indoles - therapeutic use
/ Life Sciences
/ Male
/ Mutation
/ 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
2021
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Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes
by
Moskowitz, Samuel M
, Polineni, Deepika
, Fajac, Isabelle
, Ramsey, Bonnie W
, Tullis, Elizabeth
, Ahluwalia, Neil
, Tian, Simon
, Barry, Peter J
, Álvarez, Antonio
, de Winter-de Groot, Karin M
, Mall, Marcus A
, Taylor-Cousar, Jennifer L
, Zhang, Yaohua
, Waltz, David
, Xuan, Fengjuan
, Rowe, Steven M
, Colombo, Carla
, Jun, Lucy S
, Sutharsan, Sivagurunathan
, McBennett, Kimberly A
, McKone, Edward F
, Prieto-Centurion, Valentin
in
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
/ Female
/ Gating
/ Genetics
/ Genetics General
/ Genotype
/ Genotypes
/ Human health and pathology
/ Humans
/ Indoles
/ Indoles - adverse effects
/ Indoles - therapeutic use
/ Life Sciences
/ Male
/ Mutation
/ 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
2021
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Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes
Journal Article
Triple Therapy for Cystic Fibrosis Phe508del–Gating and –Residual Function Genotypes
2021
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Overview
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.
Publisher
Massachusetts Medical Society
Subject
/ Adult
/ Alleles
/ Aminophenols - adverse effects
/ Aminophenols - therapeutic use
/ Benzodioxoles - adverse effects
/ Benzodioxoles - therapeutic use
/ Child
/ Chloride
/ Chloride Channel Agonists - adverse effects
/ Chloride Channel Agonists - therapeutic use
/ COVID-19
/ Cystic Fibrosis - drug therapy
/ Cystic Fibrosis Transmembrane Conductance Regulator
/ Cystic Fibrosis Transmembrane Conductance Regulator - genetics
/ Design
/ Female
/ Gating
/ Genetics
/ Genotype
/ Humans
/ Indoles
/ Male
/ Mutation
/ Patients
/ Proteins
/ Quinolines - adverse effects
/ Quinolines - therapeutic use
/ Sweat
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