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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: brooks pj. Nat Rev Drug Discov. 2024 Nov 4. doi: 10.1038/s41573-024-01059-3. Online ahead of print. Nat Rev Drug Discov. 2024. PMID: 39496921 Review.
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: brooks pj. 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.
Targeting shared molecular etiologies to accelerate drug development for rare diseases.
Zanello G, Garrido-Estepa M, Crespo A, O'Connor D, Nabbout R, Waters C, Hall A, Taglialatela M, Chan CH, Pearce DA, Dooms M, Brooks PJ. Zanello G, et al. Among authors: brooks pj. EMBO Mol Med. 2023 Jul 10;15(7):e17159. doi: 10.15252/emmm.202217159. Epub 2023 Jun 27. EMBO Mol Med. 2023. PMID: 37366158 Free PMC article. Review.
Gene-targeted therapies: Overview and implications.
Brooks PJ, Urv TK, Parisi MA. Brooks PJ, et al. Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):13-18. doi: 10.1002/ajmg.c.32033. Epub 2023 Mar 2. Am J Med Genet C Semin Med Genet. 2023. PMID: 36864710 Free PMC article.
211 results