Kimberly McBennett

Triple Therapy for Cystic Fibrosis <i>Phe508del</i> –Gating and –Residual Function Genotypes

Peter J. Barry, Marcus Mall, Antonio Álvarez et al. · New England Journal of Medicine · 2021 · 275 citations · Full text

Life Sciences, Genetics, Pharmacotherapy +16

Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double‐blind, placebo‐controlled phase 1/2 clinical study

Steven M. Rowe, Jonathan B. Zuckerman, Daniel Dorgan et al. · Journal of Cystic Fibrosis · 2023 · 135 citations · Full text

Pharmacy, Pharmacology, Pharmacotherapy +10

Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials

Nicole Mayer-Hamblett, Félix Ratjen, Renee Russell et al. · The Lancet Respiratory Medicine · 2022 · 126 citations · Full text

Pharmacy, Pharmacotherapy, Good Clinical Practice +8

Long-term safety and efficacy of tezacaftor–ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study

Patrick A. Flume, Reta Fischer Biner, D.G. Downey et al. · The Lancet Respiratory Medicine · 2021 · 51 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +13

Lumacaftor/ivacaftor therapy is associated with reduced hepatic steatosis in cystic fibrosis patients

Katherine Kutney, Shannon Donnola, Chris A. Flask et al. · World Journal of Hepatology · 2019 · 47 citations · Full text

Medicine, Endocrinology, Internal Medicine +14