PUBLICATIONS

Publications on Orphan Drugs

  1. 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.
  2. 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.
  3. 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 .
  4. 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.
  5. 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 .
  6. 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 .
  7. 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 .
  8. Subbiah, V, Othus, M, Palma, J, Cuglievan, B, Kurzrock, R. Designing Clinical Trials for Patients With Rare Cancers: Connecting the Zebras. Am Soc Clin Oncol Educ Book. 2025;45 (3):e100051. doi: 10.1200/EDBK-25-100051. PubMed PMID:40228175 .
  9. Hollak, CEM, Reparon-Schuijt, CC, Verdeyen, K, Deesker, LJ, Groothoff, JW, Vos, JMI et al.. New opportunities for accessing promising non-oncological orphan drugs. Lancet Reg Health Eur. 2025;52 :101275. doi: 10.1016/j.lanepe.2025.101275. PubMed PMID:40224369 PubMed Central PMC11987665.
  10. Haigo, H, Matsuda, K, Shikano, M. Clinical data required for the approval of pediatric pharmaceuticals in Japan. BMC Pediatr. 2025;25 (1):289. doi: 10.1186/s12887-025-05646-0. PubMed PMID:40223100 PubMed Central PMC11995577.
  11. Xu, L, Chen, S, Fu, W, Lin, X, Zhang, F, Qin, G et al.. Environmental toxicant 2,3,7,8-tetrachlorodibenzo-p-dioxin induces non-obstructive azoospermia: New insights from network toxicology, integrated machine learning, and biomolecular modeling. Ecotoxicol Environ Saf. 2025;295 :118173. doi: 10.1016/j.ecoenv.2025.118173. PubMed PMID:40215689 .
  12. Price, ML, Wyatt, RA, Correia, J, Areej, Z, Hinds, M, Crastin, A et al.. Identification of anti-resorptive GPCRs by high-content imaging in human osteoclasts. J Mol Endocrinol. 2025;74 (4):. doi: 10.1530/JME-24-0143. PubMed PMID:40215127 .
  13. Lexchin, J. Time to completion of conditions required by Health Canada after approving new drugs: A cohort study. Health Policy. 2025;155 :105314. doi: 10.1016/j.healthpol.2025.105314. PubMed PMID:40209308 .
  14. Dumbuya, JS, Zeng, C, Deng, L, Li, Y, Chen, X, Ahmad, B et al.. The impact of rare diseases on the quality of life in paediatric patients: current status. Front Public Health. 2025;13 :1531583. doi: 10.3389/fpubh.2025.1531583. PubMed PMID:40196857 PubMed Central PMC11973084.
  15. Le, DQ, Burton, BN, Tejeda, CJ, Hong, JC, Lee, JS, Kamdar, N et al.. Drug Price and Health Policy Knowledge Influence Prescription Behavior in Orphan Diseases: Pheochromocytoma and Paraganglioma As Prototypes of Orphan Drug Econometrics. Cureus. 2025;17 (3):e80156. doi: 10.7759/cureus.80156. PubMed PMID:40196061 PubMed Central PMC11973530.
  16. Roman, YM. Pharmacogenomics and rare diseases: optimizing drug development and personalized therapeutics. Pharmacogenomics. 2025; :1-8. doi: 10.1080/14622416.2025.2490465. PubMed PMID:40194983 .
  17. Koong, AJ, Irvin, VL, Narayan, A, Song, S, Kaplan, RM. Evidence available and used by the Food and Drug Administration for the approval of orphan and nonorphan drugs. Health Aff Sch. 2025;3 (4):qxaf057. doi: 10.1093/haschl/qxaf057. PubMed PMID:40190699 PubMed Central PMC11970248.
  18. Zhao, Y, Yue, D, Zou, Y. Orphan G Protein-Coupled Receptor GPR88: Mapping Its Significance in Central Nervous System Disorders. Mol Neurobiol. 2025; :. doi: 10.1007/s12035-025-04893-7. PubMed PMID:40184024 .
  19. El-Sayed, AA, Reiss, UM, Hanna, D, Bolous, NS. The role of public health in rare diseases: hemophilia as an example. Front Public Health. 2025;13 :1450625. doi: 10.3389/fpubh.2025.1450625. PubMed PMID:40182514 PubMed Central PMC11965367.
  20. Climente Martí, M, Edo Solsona, MD, Abad Sazatornil, MR, Bachiller Cacho, MP, Fernández Espinola, S, Guijarro Martínez, P et al.. OrPhar-SEFH 2024-2027 Strategic Plan. Farm Hosp. 2025; :. doi: 10.1016/j.farma.2025.03.001. PubMed PMID:40180861 .
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