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2010 1
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67 results

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Page 1
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.
Deep Phenotyping of Parkinson's Disease.
Dorsey ER, Omberg L, Waddell E, Adams JL, Adams R, Ali MR, Amodeo K, Arky A, Augustine EF, Dinesh K, Hoque ME, Glidden AM, Jensen-Roberts S, Kabelac Z, Katabi D, Kieburtz K, Kinel DR, Little MA, Lizarraga KJ, Myers T, Riggare S, Rosero SZ, Saria S, Schifitto G, Schneider RB, Sharma G, Shoulson I, Stevenson EA, Tarolli CG, Luo J, McDermott MP. Dorsey ER, et al. Among authors: augustine ef. J Parkinsons Dis. 2020;10(3):855-873. doi: 10.3233/JPD-202006. J Parkinsons Dis. 2020. PMID: 32444562 Free PMC article. Review.
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.
The state-of-the-art of N-of-1 therapies and the IRDiRC N-of-1 development roadmap.
Jonker AH, Tataru EA, Graessner H, Dimmock D, Jaffe A, Baynam G, Davies J, Mitkus S, Iliach O, Horgan R, Augustine EF, Bateman-House A, Pasmooij AMG, Yu T, Synofzik M, Douville J, Lapteva L, Brooks PJ, O'Connor D, Aartsma-Rus A; N-of-1 Task Force of the International Rare Diseases Research Consortium (IRDiRC). Jonker AH, et al. Among authors: augustine ef. Nat Rev Drug Discov. 2025 Jan;24(1):40-56. doi: 10.1038/s41573-024-01059-3. Epub 2024 Nov 4. Nat Rev Drug Discov. 2025. PMID: 39496921 Review.
67 results