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Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, Pendergast MK, Goldkind SF, Lee EA, Kuniholm A, Soucy A, Vaze J, Belur NR, Fredriksen K, Stojkovska I, Tsytsykova A, Armant M, DiDonato RL, Choi J, Cornelissen L, Pereira LM, Augustine EF, Genetti CA, Dies K, Barton B, Williams L, Goodlett BD, Riley BL, Pasternak A, Berry ER, Pflock KA, Chu S, Reed C, Tyndall K, Agrawal PB, Beggs AH, Grant PE, Urion DK, Snyder RO, Waisbren SE, Poduri A, Park PJ, Patterson A, Biffi A, Mazzulli JR, Bodamer O, Berde CB, Yu TW. Kim J, et al. Among authors: augustine ef. N Engl J Med. 2019 Oct 24;381(17):1644-1652. doi: 10.1056/NEJMoa1813279. Epub 2019 Oct 9. N Engl J Med. 2019. PMID: 31597037 Free PMC article.
Teacher Knowledge of Tourette Syndrome and Associated Factors.
Sapozhnikov Y, Mink JW, Augustine EF, Adams HR, Vierhile A, Lewin AB, Collins AT, McDermott MP, O'Connor T, Kurlan R, Murphy TK, Vermilion J. Sapozhnikov Y, et al. Among authors: augustine ef. Pediatr Neurol. 2023 Aug;145:80-87. doi: 10.1016/j.pediatrneurol.2023.05.007. Epub 2023 May 16. Pediatr Neurol. 2023. PMID: 37295059
Data sharing to advance gene-targeted therapies in rare diseases.
Lekstrom-Himes J, Augustine EF, Brower A, Defay T, Finkel RS, McGuire AL, Skinner MW, Yu TW. Lekstrom-Himes J, et al. Among authors: augustine ef. Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):87-98. doi: 10.1002/ajmg.c.32028. Epub 2023 Jan 3. Am J Med Genet C Semin Med Genet. 2023. PMID: 36594517
Batten disease: an expert update on agents in preclinical and clinical trials.
Masten MC, Mink JW, Augustine EF. Masten MC, et al. Among authors: augustine ef. Expert Opin Investig Drugs. 2020 Dec;29(12):1317-1322. doi: 10.1080/13543784.2020.1837110. Epub 2020 Nov 1. Expert Opin Investig Drugs. 2020. PMID: 33135495 Free PMC article. No abstract available.
Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies.
Lekstrom-Himes J, Brooks PJ, Koeberl DD, Brower A, Goldenberg A, Green RC, Morris JA, Orsini JJ, Yu TW, Augustine EF. Lekstrom-Himes J, et al. Among authors: augustine ef. Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):30-43. doi: 10.1002/ajmg.c.32031. Epub 2023 Feb 4. Am J Med Genet C Semin Med Genet. 2023. PMID: 36738469 Free PMC article.
69 results