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Journal of Comparative Effectiveness Research|October 25, 2021
Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophyPerry B Shieh, Gary Elfring, Panayiota Trifillis, et al.Journal of Applied Physiology (Bethesda, Md. : 1985)|June 19, 2010
Membrane blebbing as an assessment of functional rescue of dysferlin-deficient human myotubes via nonsense suppressionBingjing Wang, Zhaohui Yang, Becky K Brisson, et al.Plos One|December 19, 2013
Phase 2a study of ataluren-mediated dystrophin production in patients with nonsense mutation Duchenne muscular dystrophyRichard S Finkel, Kevin M Flanigan, Brenda Wong, et al.Journal of Clinical Pharmacology|March 29, 2007
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteersSamit Hirawat, Ellen M Welch, Gary L Elfring, et al.Muscle & Nerve|October 11, 2019
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trialsCraig M McDonald, Gautam Sajeev, Zhiwen Yao, et al.Muscle & Nerve|May 16, 2013
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter studyCraig M McDonald, Erik K Henricson, R Ted Abresch, et al.Clinical Pharmacology in Drug Development|June 17, 2016
Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein TranslationMarla Weetall, Thomas Davis, Gary Elfring, et al.Journal of Cystic Fibrosis : Official Journal of the European Cystic Fibrosis Society|April 5, 2011
Ambulatory quantitative waking and sleeping cough assessment in patients with cystic fibrosisEitan Kerem, Michael Wilschanski, Nilsen L Miller, et al.The Journal of Biological Chemistry|April 19, 2011
High-throughput screening uncovers a compound that activates latent HIV-1 and acts cooperatively with a histone deacetylase (HDAC) inhibitorSofiya Micheva-Viteva, Yoshifumi Kobayashi, Leonard C Edelstein, et al.Plos One|December 20, 2016
Discovery of Novel Small Molecule Inhibitors of VEGF Expression in Tumor Cells Using a Cell-Based High Throughput Screening PlatformLiangxian Cao, Marla Weetall, Jenelle Bombard, et al.Pageof 5