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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: o connor d. 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.
Research on rare diseases: ten years of progress and challenges at IRDiRC.
Monaco L, Zanello G, Baynam G, Jonker AH, Julkowska D, Hartman AL, O'Connor D, Wang CM, Wong-Rieger D, Pearce DA. Monaco L, et al. Nat Rev Drug Discov. 2022 May;21(5):319-320. doi: 10.1038/d41573-022-00019-z. Nat Rev Drug Discov. 2022. PMID: 35079160 Free PMC article. No abstract available.
Defining rare conditions in the era of personalized medicine.
O'Connor DJ, Gabaldo M, Aartsma-Rus A, Hechtelt Jonker A. O'Connor DJ, et al. Nat Rev Drug Discov. 2023 Nov;22(11):857-858. doi: 10.1038/d41573-023-00145-2. Nat Rev Drug Discov. 2023. PMID: 37684343 No abstract available.
Assessment of significant benefit for orphan medicinal products by European regulators may support subsequent relative effectiveness assessments by health technology assessment organizations.
Vreman RA, de Ruijter AS, Zawada A, Tafuri G, Stoyanova-Beninska V, O'Connor D, Naumann-Winter F, Wolter F, Mantel-Teeuwisse AK, Leufkens HGM, Sidiropoulos I, Larsson K, Goettsch WG. Vreman RA, et al. Drug Discov Today. 2020 Jul;25(7):1223-1231. doi: 10.1016/j.drudis.2020.04.012. Epub 2020 Apr 25. Drug Discov Today. 2020. PMID: 32344040 Free article. Review.
A Hearing Intervention and Health-Related Quality of Life in Older Adults: A Secondary Analysis of the ACHIEVE Randomized Clinical Trial.
Huang AR, Morales EG, Arnold ML, Burgard S, Couper D, Deal JA, Glynn NW, Gmelin T, Goman AM, Gravens-Mueller L, Hayden KM, Mitchell CM, Pankow JS, Pike JR, Reed NS, Sanchez VA, Schrack JA, Sullivan KJ, Coresh J, Lin FR, Chisolm TH; ACHIEVE Collaborative Research Group. Huang AR, et al. JAMA Netw Open. 2024 Nov 4;7(11):e2446591. doi: 10.1001/jamanetworkopen.2024.46591. JAMA Netw Open. 2024. PMID: 39570588 Free PMC article. Clinical Trial.
Unlocking Engagement: Enhancing Participation in Research With Vulnerable Populations.
Rojas-Rozo L, Arsenault-Lapierre G, Dumaresq D, Trépanier T, Lea P, Myers Barnett K, O'Connor D, Fernandez Loughlin R, Miskucza K, Wighton MB, Godard-Sebillotte C, Gruneir A, Beuscart JB, Bronskill SE, Sourial N, Smith EE, Bethell J, Vedel I; COVID ROSA Research Team. Rojas-Rozo L, et al. Among authors: o connor d. Int J Public Health. 2024 Oct 31;69:1606705. doi: 10.3389/ijph.2024.1606705. eCollection 2024. Int J Public Health. 2024. PMID: 39568528 Free PMC article. No abstract available.
2,784 results