Publications on Orphan Drugs

  1. Cooner, F, Ye, J, Reaman, G. Clinical trial considerations for pediatric cancer drug development. J Biopharm Stat. 2023; :1-16. doi: 10.1080/10543406.2023.2172424. PubMed PMID:36749066 .
  2. Decker, B, Mlcoch, T, Pustovalova, A, Dolezal, T. Novel approach to decision making for orphan drugs. Int J Technol Assess Health Care. 2023;39 (1):e10. doi: 10.1017/S0266462323000053. PubMed PMID:36748356 .
  3. Wong, TS, Gao, W, Chen, G, Qiu, C, He, G, Ye, F et al.. Cryo-EM structure of orphan G protein-coupled receptor GPR21. MedComm (2020). 2023;4 (1):e205. doi: 10.1002/mco2.205. PubMed PMID:36721851 PubMed Central PMC9877262.
  4. Kari, S, Murugesan, A, Thiyagarajan, R, Kidambi, S, Razzokov, J, Selvaraj, C et al.. Bias-force guided simulations combined with experimental validations towards GPR17 modulators identification. Biomed Pharmacother. 2023;160 :114320. doi: 10.1016/j.biopha.2023.114320. PubMed PMID:36716660 .
  5. Brown, DG, Wobst, HJ. Analysis of orphan designation status for FDA approved drugs, and case studies in oncology, neuroscience and metabolic diseases. Bioorg Med Chem. 2023;80 :117170. doi: 10.1016/j.bmc.2023.117170. PubMed PMID:36696875 .
  6. Jenei, K, Gyawali, B. Expensive Drug Prices for Rare Cancers: Are Patients Truly Benefitting?. Healthc Pap. 2023;21 (1):44-51. doi: 10.12927/hcpap.2023.26996. PubMed PMID:36692916 .
  7. Keating, B. What Procedural Ethics Can Learn from the Quest for Moral Justification for the "Rule of Rescue". Healthc Pap. 2023;21 (1):38-43. doi: 10.12927/hcpap.2023.26997. PubMed PMID:36692915 .
  8. Vassal, G, de Rojas, T, Pearson, ADJ. Impact of the EU Paediatric Medicine Regulation on new anti-cancer medicines for the treatment of children and adolescents. Lancet Child Adolesc Health. 2023; :. doi: 10.1016/S2352-4642(22)00344-3. PubMed PMID:36682367 .
  9. Zhan, N, Wang, B, Martens, N, Liu, Y, Zhao, S, Voortman, G et al.. Identification of Side Chain Oxidized Sterols as Novel Liver X Receptor Agonists with Therapeutic Potential in the Treatment of Cardiovascular and Neurodegenerative Diseases. Int J Mol Sci. 2023;24 (2):. doi: 10.3390/ijms24021290. PubMed PMID:36674804 PubMed Central PMC9863018.
  10. Benedetto Tiz, D, Bagnoli, L, Rosati, O, Marini, F, Sancineto, L, Santi, C et al.. Top Selling (2026) Small Molecule Orphan Drugs: A Journey into Their Chemistry. Int J Mol Sci. 2023;24 (2):. doi: 10.3390/ijms24020930. PubMed PMID:36674441 PubMed Central PMC9864910.
  11. Tian, SY, Chen, SM, Feng, YY, He, JL, Li, Y. Ginseng-derived panaxadiol ameliorates STZ-induced type 1 diabetes through inhibiting RORγ/IL-17A axis. Acta Pharmacol Sin. 2023; :. doi: 10.1038/s41401-022-01042-x. PubMed PMID:36650291 .
  12. de Rojas, T, Kearns, P, Blanc, P, Skolnik, J, Fox, E, Knox, L et al.. Changing incentives to ACCELERATE drug development for paediatric cancer. Cancer Med. 2023; :. doi: 10.1002/cam4.5627. PubMed PMID:36645217 .
  13. Aiello, A, Mancuso, ME, Leone, S, Rossi, L, Cioni, L, Teruzzi, C et al.. Cost analysis of extended half-life recombinant factor IX products in the treatment of haemophilia B in Italy: an update. Glob Reg Health Technol Assess. 2022;9 :9-13. doi: 10.33393/grhta.2022.2331. PubMed PMID:36628322 PubMed Central PMC9768593.
  14. Na, SY, Kim, KS, Jung, YS, Kim, DK, Kim, J, Cho, SJ et al.. An Inverse Agonist GSK5182 Increases Protein Stability of the Orphan Nuclear Receptor ERRγ via Inhibition of Ubiquitination. Int J Mol Sci. 2022;24 (1):. doi: 10.3390/ijms24010096. PubMed PMID:36613556 PubMed Central PMC9820335.
  15. Stafinski, T, Street, J, Young, A, Menon, D. Moving beyond the Court of Public Opinion: A Citizens' Jury Exploring the Public's Values around Funding Decisions for Ultra-Orphan Drugs. Int J Environ Res Public Health. 2022;20 (1):. doi: 10.3390/ijerph20010633. PubMed PMID:36612953 PubMed Central PMC9819519.
  16. Zhang, M, Shi, Z, Zhang, S, Li, X, To, SKY, Peng, Y et al.. The Ginsenoside Compound K Suppresses Stem-Cell-like Properties and Colorectal Cancer Metastasis by Targeting Hypoxia-Driven Nur77-Akt Feed-Forward Signaling. Cancers (Basel). 2022;15 (1):. doi: 10.3390/cancers15010024. PubMed PMID:36612021 PubMed Central PMC9817892.
  17. Poveda, JL, Gómez, C, Gil, A, Badia, X. ODs with a positive TPR conclusion, not subject to a conditional approval, and approved without requering a pass would be more likely to be reimbursed in Spain. Orphanet J Rare Dis. 2023;18 (1):4. doi: 10.1186/s13023-022-02610-4. PubMed PMID:36609401 PubMed Central PMC9824902.
  18. Ranganath, LR, Sireau, N. Clinical development innovation in rare diseases: overcoming barriers to successful delivery of a randomised clinical trial in alkaptonuria-a mini-review. Orphanet J Rare Dis. 2023;18 (1):1. doi: 10.1186/s13023-022-02606-0. PubMed PMID:36600285 PubMed Central PMC9811731.
  19. Gahbauer, S, Correy, GJ, Schuller, M, Ferla, MP, Doruk, YU, Rachman, M et al.. Iterative computational design and crystallographic screening identifies potent inhibitors targeting the Nsp3 macrodomain of SARS-CoV-2. Proc Natl Acad Sci U S A. 2023;120 (2):e2212931120. doi: 10.1073/pnas.2212931120. PubMed PMID:36598939 .
  20. Akinosoglou, K, Kotsaki, A, Gounaridi, IM, Christaki, E, Metallidis, S, Adamis, G et al.. Efficacy and safety of early soluble urokinase plasminogen receptor plasma-guided anakinra treatment of COVID-19 pneumonia: A subgroup analysis of the SAVE-MORE randomised trial. EClinicalMedicine. 2023;56 :101785. doi: 10.1016/j.eclinm.2022.101785. PubMed PMID:36590789 PubMed Central PMC9791950.
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