PUBLICATIONS

Publications on Orphan Drugs

  1. Shanmugaraj, B, Ravi, K, Baladevan, K. Plant molecular farming: a promising frontier for orphan drug production. Biotechnol Lett. 2025;47 (3):56. doi: 10.1007/s10529-025-03596-2. PubMed PMID:40381123 .
  2. Abozaid, GM, Al-Omar, HA, Alrabiah, A, AlMuaither, A, McKnight, AJ. Towards a sustainable rare disease and orphan drug ecosystem in Saudi Arabia: policy insights from a multi-stakeholder workshop. Front Pharmacol. 2025;16 :1583477. doi: 10.3389/fphar.2025.1583477. PubMed PMID:40376269 PubMed Central PMC12078156.
  3. Chippi, C. Amgen Enablement and its Undue Burden on an Overburdened Biomedical Industry. J Law Health. 2025;38 (3):383-401. . PubMed PMID:40338962 .
  4. Zhang, L, Gatlin, V, Gupta, S, Salinas, ML, Romero, S, Cai, JJ et al.. Expression of Prooncogenic Nuclear Receptor 4A (NR4A)-Regulated Genes β1-Integrin and G9a Inhibited by Dual NR4A1/2 Ligands. Int J Mol Sci. 2025;26 (8):. doi: 10.3390/ijms26083909. PubMed PMID:40332813 PubMed Central PMC12028307.
  5. Shao, Y, Mei, Y, Tan, Y, Yang, M, Wu, H. The regulatory functions of G protein-coupled receptors signaling pathways in B cell differentiation and development contributing to autoimmune diseases. Cell Biosci. 2025;15 (1):57. doi: 10.1186/s13578-025-01398-7. PubMed PMID:40307944 PubMed Central PMC12042570.
  6. Awada, N. Access to drugs for rare diseases (DRD) in Canada: a comprehensive review of the provincial DRD-specific programs. Ther Adv Rare Dis. 2025;6 :26330040251335660. doi: 10.1177/26330040251335660. PubMed PMID:40302781 PubMed Central PMC12038190.
  7. Young, CM, Phares, SE, Kennedy, A, Sullivan, J, McGowan, B, Trusheim, MR et al.. Pediatric-onset rare disease therapy pipeline yields hope for some and gaps for many: 10-year projection of approvals, treated patients, and list price revenues. J Manag Care Spec Pharm. 2025;31 (5):491-498. doi: 10.18553/jmcp.2025.31.5.491. PubMed PMID:40298308 PubMed Central PMC12039501.
  8. Russell, IC, Lee, D, Wootten, D, Sexton, PM, Bumbak, F. Cryoelectron microscopy as a tool for illuminating activation mechanisms of human class A orphan G protein-coupled receptors. Pharmacol Rev. 2025;77 (3):100056. doi: 10.1016/j.pharmr.2025.100056. PubMed PMID:40286430 .
  9. Chen, T, An, Q, Zhang, T, Yan, S, Li, J, Song, X et al.. Mechanism of Penoxsulam's Effect on Chlorophyll Synthesis and the Metabolism of Foxtail Millet. Plants (Basel). 2025;14 (8):. doi: 10.3390/plants14081211. PubMed PMID:40284098 PubMed Central PMC12030371.
  10. Cooper, N, Guterres, S, Pochopień, M, Wilson, K, James, S, Toumi, M et al.. The Cost-Effectiveness of Avatrombopag Versus Eltrombopag and Romiplostim in the Treatment of Patients with Immune Thrombocytopenia in the UK. J Mark Access Health Policy. 2025;13 (2):11. doi: 10.3390/jmahp13020011. PubMed PMID:40276091 PubMed Central PMC12015888.
  11. Xiao, J, Meng, Z, Lu, Y, Nie, Z, Liu, Y, Yao, Z et al.. Targeting microglia-Th17 feed-forward loop to suppress autoimmune neuroinflammation. J Neuroinflammation. 2025;22 (1):118. doi: 10.1186/s12974-025-03427-1. PubMed PMID:40275354 PubMed Central PMC12023695.
  12. Sauerbruch, T, Aretz, S, Hebestreit, H, Kaemmerer, H, Nährlich, L, Siegmund, B et al.. [Rare diseases: the role of internal medicine]. Inn Med (Heidelb). 2025;66 (5):533-539. doi: 10.1007/s00108-025-01892-7. PubMed PMID:40272470 PubMed Central PMC12053190.
  13. Brown, F, Vargas, M, Stanisic, S, Fatzinger, G, Iliach, O. Impact of changes in regulatory framework on approval of medicines for rare diseases and applicability to market access policies. Front Med (Lausanne). 2025;12 :1474087. doi: 10.3389/fmed.2025.1474087. PubMed PMID:40270510 PubMed Central PMC12016575.
  14. Ahmed, MA, AbuAsal, B, Barrett, JS, Azer, K, Hon, YY, Albusaysi, S et al.. Unlocking the Mysteries of Rare Disease Drug Development: A Beginner's Guide for Clinical Pharmacologists. Clin Transl Sci. 2025;18 (4):e70215. doi: 10.1111/cts.70215. PubMed PMID:40261641 PubMed Central PMC12013510.
  15. Ida, T, Matsui, K, Nagata, S, Nakamachi, T, Shiimura, Y, Sato, T et al.. Discovery of Feeding Regulatory Peptides and The Importance of Peptide Discovery Research. Kurume Med J. 2025; :. doi: 10.2739/kurumemedj.MS7134001. PubMed PMID:40254448 .
  16. Sankhe, S, Fofana, FG, Heiba, W, Ogunyemi, OM, Masisi, K, Muiruri, I et al.. BioStruct-Africa's capacity building workshops as a model for advancing the emerging community of structural biologists in Africa. Commun Chem. 2025;8 (1):118. doi: 10.1038/s42004-025-01519-w. PubMed PMID:40253564 PubMed Central PMC12009398.
  17. Antigny, F, Luo, R, Perrier, R, Masson, B, Fadel, G, Ruffenach, G et al.. Ca2+ Cycling Alteration in a Porcine Model of Right Ventricular Dysfunction. Circ Heart Fail. 2025;18 (5):e012293. doi: 10.1161/CIRCHEARTFAILURE.124.012293. PubMed PMID:40248873 PubMed Central PMC12084022.
  18. Ma, C, Sun, H, Shen, C, Li, X, Shen, Y. Discovery of a first-in-class protein arginine methyltransferase 1 (PRMT1) degrader for nonenzymatic functions studies. Eur J Med Chem. 2025;291 :117625. doi: 10.1016/j.ejmech.2025.117625. PubMed PMID:40245820 .
  19. Kinzel, O, Goldberg, SD, Cummings, MD, Gege, C, Steeneck, C, Xue, X et al.. Identification of JNJ-61803534, a RORγt Inverse Agonist for the Treatment of Psoriasis. J Med Chem. 2025;68 (8):8713-8728. doi: 10.1021/acs.jmedchem.5c00390. PubMed PMID:40237323 .
  20. Risser, LM, Witte, T, Englbrecht, M, Strunz, PP, Froehlich, M, Schmalzing, M et al.. Janus kinase inhibitors show a longer drug survival than biologics in a real-world cohort of patients with rheumatoid arthritis - a retrospective analysis from the RHADAR database. Rheumatol Int. 2025;45 (5):100. doi: 10.1007/s00296-025-05859-7. PubMed PMID:40232336 .
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