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Antimicrobial Agents and Chemotherapy|May 1, 2013
Identification of PTC725, an orally bioavailable small molecule that selectively targets the hepatitis C Virus NS4B proteinZhengxian Gu, Jason D Graci, Frederick C Lahser, et al.Plos One|October 26, 2018
The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potentialWestley J Friesen, Briana Johnson, Jairo Sierra, et al.RNA (New York, N.Y.)|January 19, 2017
The nucleoside analog clitocine is a potent and efficacious readthrough agentWestley J Friesen, Christopher R Trotta, Yuki Tomizawa, et al.Virus Research|November 29, 2020
The DHODH inhibitor PTC299 arrests SARS-CoV-2 replication and suppresses induction of inflammatory cytokinesJeremy Luban, Rachel A Sattler, Elke Mühlberger, et al.Nature Communications|December 16, 2021
Small molecule splicing modifiers with systemic HTT-lowering activityAnuradha Bhattacharyya, Christopher R Trotta, Jana Narasimhan, et al.The Lancet. Respiratory Medicine|May 20, 2014
Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trialEitan Kerem, Michael W Konstan, Kris De Boeck, et al.Molecular Cancer Therapeutics|October 25, 2018
Targeting of Hematologic Malignancies with PTC299, A Novel Potent Inhibitor of Dihydroorotate Dehydrogenase with Favorable Pharmaceutical PropertiesLiangxian Cao, Marla Weetall, Christopher Trotta, et al.Lancet (London, England)|July 22, 2017
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trialCraig M McDonald, Craig Campbell, Ricardo Erazo Torricelli, et al.Muscle & Nerve|July 22, 2014
Ataluren treatment of patients with nonsense mutation dystrophinopathyKatharine Bushby, Richard Finkel, Brenda Wong, et al.Journal of Comparative Effectiveness Research|August 28, 2020
Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophyCraig Campbell, Richard J Barohn, Enrico Bertini, et al.Pageof 5