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Phase 1 Study of Safety, Tolerability, and Pharmacokinetics of PTC299, an Inhibitor of Stress-Regulated Protein Translation.
Weetall M, Davis T, Elfring G, Northcutt V, Cao L, Moon YC, Riebling P, Dali M, Hirawat S, Babiak J, Colacino J, Almstead N, Spiegel R, Peltz SW. Weetall M, et al. Among authors: elfring g. Clin Pharmacol Drug Dev. 2016 Jul;5(4):296-305. doi: 10.1002/cpdd.240. Epub 2016 Feb 2. Clin Pharmacol Drug Dev. 2016. PMID: 27310330 Free PMC article. Clinical Trial.
Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.
Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL. Hirawat S, et al. Among authors: elfring gl. J Clin Pharmacol. 2007 Apr;47(4):430-44. doi: 10.1177/0091270006297140. J Clin Pharmacol. 2007. PMID: 17389552 Clinical Trial.
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E; Clinical Evaluator Training Group; ACT DMD Study Group. McDonald CM, et al. Among authors: elfring g. Lancet. 2017 Sep 23;390(10101):1489-1498. doi: 10.1016/S0140-6736(17)31611-2. Epub 2017 Jul 17. Lancet. 2017. PMID: 28728956 Clinical Trial.
The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.
Friesen WJ, Johnson B, Sierra J, Zhuo J, Vazirani P, Xue X, Tomizawa Y, Baiazitov R, Morrill C, Ren H, Babu S, Moon YC, Branstrom A, Mollin A, Hedrick J, Sheedy J, Elfring G, Weetall M, Colacino JM, Welch EM, Peltz SW. Friesen WJ, et al. Among authors: elfring g. PLoS One. 2018 Oct 25;13(10):e0206158. doi: 10.1371/journal.pone.0206158. eCollection 2018. PLoS One. 2018. PMID: 30359426 Free PMC article.
Deflazacort versus prednisone/prednisolone for maintaining motor function and delaying loss of ambulation: A post HOC analysis from the ACT DMD trial.
Shieh PB, Mcintosh J, Jin F, Souza M, Elfring G, Narayanan S, Trifillis P, Peltz SW, Mcdonald CM, Darras BT; THE ACT DMD STUDY GROUP. Shieh PB, et al. Among authors: elfring g. Muscle Nerve. 2018 Nov;58(5):639-645. doi: 10.1002/mus.26191. Epub 2018 Sep 27. Muscle Nerve. 2018. PMID: 30028519 Free PMC article. Clinical Trial.
Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.
Kerem E, Konstan MW, De Boeck K, Accurso FJ, Sermet-Gaudelus I, Wilschanski M, Elborn JS, Melotti P, Bronsveld I, Fajac I, Malfroot A, Rosenbluth DB, Walker PA, McColley SA, Knoop C, Quattrucci S, Rietschel E, Zeitlin PL, Barth J, Elfring GL, Welch EM, Branstrom A, Spiegel RJ, Peltz SW, Ajayi T, Rowe SM; Cystic Fibrosis Ataluren Study Group. Kerem E, et al. Lancet Respir Med. 2014 Jul;2(7):539-47. doi: 10.1016/S2213-2600(14)70100-6. Epub 2014 May 15. Lancet Respir Med. 2014. PMID: 24836205 Free PMC article. Clinical Trial.
Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.
McDonald CM, Sajeev G, Yao Z, McDonnell E, Elfring G, Souza M, Peltz SW, Darras BT, Shieh PB, Cox DA, Landry J, Signorovitch J; ACT DMD Study Group and the Tadalafil DMD Study Group. McDonald CM, et al. Among authors: elfring g. Muscle Nerve. 2020 Jan;61(1):26-35. doi: 10.1002/mus.26736. Epub 2019 Nov 7. Muscle Nerve. 2020. PMID: 31599456 Free PMC article.
72 results