PUBLICATIONS

Publications on Orphan Drugs

  1. Jung, MS, Choi, WY, Zhang, W, Barrera, FN, Perkins, RS. The Role of Receptor Tyrosine Kinase-like Orphan Receptor 1 (ROR1) in Cancer Stem Cell Signaling. Int J Mol Sci. 2025;26 (16):. doi: 10.3390/ijms26167828. PubMed PMID:40869147 .
  2. Fluck, C. [Gene therapy methods for rare ophthalmological diseases in the light of distributional justice-An overview]. Ophthalmologie. 2025; :. doi: 10.1007/s00347-025-02308-5. PubMed PMID:40864185 .
  3. Anguera, G, Gallego, O, Llobet, M, Berga, N, Moreno-Martinez, ME, Leon, X et al.. Opening of a phase Ib/II study to investigate the safety and efficacy of Afatinib in patients with Fanconi anemia and unresectable locally advanced or metastatic head and neck squamous cell carcinoma. BMC Cancer. 2025;25 (1):1374. doi: 10.1186/s12885-025-14619-6. PubMed PMID:40859225 .
  4. Gleeson, D, Chapman, S, McAteer, H, Qin, A, Gregory, J, Pizzato, J et al.. Janus kinase inhibitors in palmoplantar pustulosis: a mixed-methods feasibility (JAKPPPOT) trial protocol. BMJ Open. 2025;15 (8):e106361. doi: 10.1136/bmjopen-2025-106361. PubMed PMID:40840981 PubMed Central PMC12374624.
  5. Sajwani, N, Suchitha, GP, Keshava Prasad, TS, Dagamajalu, S. Drug repurposing in oncology: a path beyond the bottleneck. Med Oncol. 2025;42 (10):443. doi: 10.1007/s12032-025-02994-w. PubMed PMID:40849858 .
  6. Palandri, F, Branzanti, F, Bonifacio, M, Elli, EM, Morsia, E, Farina, M et al.. Revised "iRR6" model in intermediate-1 risk myelofibrosis patients treated with ruxolitinib. Cancer. 2025;131 (17):e70062. doi: 10.1002/cncr.70062. PubMed PMID:40839414 PubMed Central PMC12369922.
  7. Bassi, I, Jabali, A, Levin, L, Lambiase, G, Moshe, N, Farag, N et al.. A high-throughput zebrafish screen identifies novel candidate treatments for kaposiform lymphangiomatosis (KLA). J Exp Med. 2025;222 (11):. doi: 10.1084/jem.20240513. PubMed PMID:40838953 .
  8. Li, N, Yan, L, Peng, A, Fu, X, Qin, H, Yao, K et al.. Voltage-gated sodium channels in the nervous system: Molecular physiology to therapeutic interventions. Neural Regen Res. 2025; :. doi: 10.4103/NRR.NRR-D-25-00260. PubMed PMID:40808385 .
  9. Stacchiotti, S, Pantziarka, P, Leonard, H, Voltz, C, Abatedaga, L, Bouche, G et al.. How to foster new treatment development in ultra-rare tumours? Joint EMA-EORTC multi-stakeholder workshops on ultra-rare sarcomas as a model for rare cancers. Cancer Treat Rev. 2025;140 :103003. doi: 10.1016/j.ctrv.2025.103003. PubMed PMID:40789252 .
  10. Sitbon, O, Sahay, S, Escribano Subías, P, Zolty, RL, Kingrey, JF, Ryan, JJ et al.. Seralutinib for the Treatment of Pulmonary Arterial Hypertension in Adults: TORREY Open-Label Extension Study. Adv Ther. 2025; :. doi: 10.1007/s12325-025-03297-2. PubMed PMID:40788460 .
  11. Knox, RP, Sarpatwari, A. Legal challenges to the 340B drug pricing program: administration, regulation, and reform. Health Aff Sch. 2025;3 (8):qxaf125. doi: 10.1093/haschl/qxaf125. PubMed PMID:40765838 PubMed Central PMC12319670.
  12. El-Nakib, HE, Abdel Aziz, SE, Schaletzky, J, Ahmed, NS. Analytical Methodologies for Anti-Infective Orphan Drugs: A Comprehensive Review of FDA Approvals (2013-2023) Part 2. Crit Rev Anal Chem. 2025; :1-25. doi: 10.1080/10408347.2025.2541764. PubMed PMID:40758480 .
  13. Garrison, SR, Siegel, IJ, Takala, CR, Vaithilingam, SL, Yang, GW, Zoghbi, AW et al.. Bridging psychiatry and rare genetic diseases: a scoping review of therapeutic strategies and diagnostic delay paired with healthcare economic burden analysis. Orphanet J Rare Dis. 2025;20 (1):393. doi: 10.1186/s13023-025-03941-8. PubMed PMID:40750893 PubMed Central PMC12315305.
  14. Abozaid, GM, Al-Omar, HA, Alrabiah, A, Alomary, H, McKnight, AJ. Shaping national rare diseases definition in Saudi Arabia: outcome from health ecosystem multisectoral workshop. Front Pharmacol. 2025;16 :1595967. doi: 10.3389/fphar.2025.1595967. PubMed PMID:40746728 PubMed Central PMC12312012.
  15. Gomase, VS, Sharma, R, Sardana, S, Dhamane, SP. The Economics of Drug Development: A Comparison Between Orphan and Non-Orphan Drugs. Curr Drug Discov Technol. 2025; :. doi: 10.2174/0115701638379700250713040806. PubMed PMID:40739691 .
  16. Cyske, Z, Radzanowska-Alenowicz, E, Rintz, E, Gaffke, L, Pierzynowska, K. The rare disease burden: a multidimensional challenge. Acta Biochim Pol. 2025;72 :14777. doi: 10.3389/abp.2025.14777. PubMed PMID:40726965 PubMed Central PMC12301258.
  17. Lee, J, Kim, JY, Lee, JH, Lee, KH. Protocatechuic acid modulates the circadian rhythm of keratinocytes and maintains skin barrier integrity. Mol Biol Rep. 2025;52 (1):765. doi: 10.1007/s11033-025-10880-x. PubMed PMID:40721875 .
  18. Tomeczkowski, J, Heidbrede, T, Eichinger, B, Osowski, U, Leverkus, F, Schmitter, S et al.. Challenges and Criteria for Single-Arm Trials Leading to an Added Benefit in German Health Technology Assessments. Pharmacoeconomics. 2025; :. doi: 10.1007/s40273-025-01524-w. PubMed PMID:40715943 .
  19. Beinfeld, MT, LaMountain, F, Phillips, G, Hughes, T, Neumann, PJ, Chambers, JD et al.. What drug characteristics explain the wide range of manufacturer rebates?. Health Aff Sch. 2025;3 (7):qxaf132. doi: 10.1093/haschl/qxaf132. PubMed PMID:40717766 PubMed Central PMC12290394.
  20. Wei, H, Burckart, GJ, Taylor-Zapata, P, Wang, Y. Labeling Pediatric Products: BPCA Efforts and Orphan Drug Opportunities. J Pediatr Pharmacol Ther. 2025;30 (2):226-238. doi: 10.5863/1551-6776-30.2.226. PubMed PMID:40717747 PubMed Central PMC12288554.
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