PUBLICATIONS

Publications on Orphan Drugs

  1. 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 .
  2. 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 .
  3. 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.
  4. 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.
  5. Magrelli, A, O'Connor, DJ, Stoyanova-Beninska, V. Editorial: The changing focus of regulatory frameworks around the globe and the opportunities for harmonization. Front Med (Lausanne). 2025;12 :1645278. doi: 10.3389/fmed.2025.1645278. PubMed PMID:40708643 PubMed Central PMC12287046.
  6. Li, X, Winters, ND, Pandey, S, Lankford, C, Stoveken, HM, Smith, E et al.. Homeostatic scaling of dynorphin signaling by a non-canonical opioid receptor. Nat Commun. 2025;16 (1):6786. doi: 10.1038/s41467-025-62133-x. PubMed PMID:40701991 PubMed Central PMC12287315.
  7. Yokota, T, Inukai, N, Shibasaki, H, Nakamura, H, Yokota, S, Nakamura, Y et al.. Report on the rare disease consortium Japan inaugural symposium - July 18, 2023, shonan health innovation park, Japan. J Neuromuscul Dis. 2025; :22143602251356742. doi: 10.1177/22143602251356742. PubMed PMID:40696833 .
  8. Inoue, S, Ozaki, A, Hakariya, H, Zhao, J, Florez, ID, Morikawa, Y et al.. Addressing the drug development challenge for rare pediatric diseases in Japan: a case study of isotretinoin. Invest New Drugs. 2025; :. doi: 10.1007/s10637-025-01566-z. PubMed PMID:40694216 .
  9. Vieujean, S, Sands, BE, Panaccione, R, Rubin, DT, Jairath, V, Danese, S et al.. Comparison of the FDA and EMA guidance on drug development in ulcerative colitis: an expert panel review. J Crohns Colitis. 2025;19 (7):. doi: 10.1093/ecco-jcc/jjaf111. PubMed PMID:40692351 PubMed Central PMC12280173.
  10. Vukicevic, S, Peric, M, Bordukalo-Niksic, T, Blazevic, V, Durdevic, D, Ivanjko, N et al.. Time to consider fracture nonunion an orphan disease? An update into pathophysiology, epidemiology and therapeutic solutions. Eur J Trauma Emerg Surg. 2025;51 (1):255. doi: 10.1007/s00068-025-02918-3. PubMed PMID:40691747 PubMed Central PMC12279619.
  11. Panaccione, R, Feagan, BG, Afzali, A, Rubin, DT, Reinisch, W, Panés, J et al.. Efficacy and safety of intravenous induction and subcutaneous maintenance therapy with guselkumab for patients with Crohn's disease (GALAXI-2 and GALAXI-3): 48-week results from two phase 3, randomised, placebo and active comparator-controlled, double-blind, triple-dummy trials. Lancet. 2025;406 (10501):358-375. doi: 10.1016/S0140-6736(25)00681-6. PubMed PMID:40684778 .
  12. Ren, H, Tao, K, Liu, J, Zu, L, Liu, Y, Hu, Y et al.. Bioactive sesquiterpene lactones from Elephantopus scaber: semi-synthesis, target fishing, anti-malignant glioma efficacy in vitro and in vivo. Eur J Med Chem. 2025;297 :117954. doi: 10.1016/j.ejmech.2025.117954. PubMed PMID:40680455 .
  13. Leng, Y, Tang, Y, Yu, W, Hou, Y, He, H, Han, Y et al.. Insights into Support Systems for Orphan Drug Development: A Comparative Study. Ther Innov Regul Sci. 2025; :. doi: 10.1007/s43441-025-00833-8. PubMed PMID:40658166 .
  14. Jose, A, Yogeswaran, A, Fuenderich, M, Kiely, D, Sweatt, AJ, Zamanian, RT et al.. Survival Outcomes and Impact of Targeted PAH Therapy in Portopulmonary Hypertension in the PVRI GoDeep Meta-Registry. Pulm Circ. 2025;15 (3):e70121. doi: 10.1002/pul2.70121. PubMed PMID:40636151 PubMed Central PMC12237829.
  15. Debnath, A, Mazumder, R, Mazumder, A, Tyagi, PK, Singh, RK. Challenges and Progress of Orphan Drug Development for Rare Diseases. Curr Pharm Biotechnol. 2025; :. doi: 10.2174/0113892010371761250616112614. PubMed PMID:40600560 .
  16. Cheng, S, Cui, L, Chen, J, Xiu, Q, Si, R, Yang, X et al.. SIGIRR: An Orphan Receptor Mediating Anti-inflammatory Actions. Expert Rev Mol Med. 2025;27 :e24. doi: 10.1017/erm.2025.10009. PubMed PMID:40583347 .
  17. Authier, S, Delatte, MS, Wakeford, A, Halpern, W, McMaster, O, Mellon, D et al.. Toxicology and safety pharmacology investigations on the nervous system: 2024 industry survey. J Pharmacol Toxicol Methods. 2025;135 :108378. doi: 10.1016/j.vascn.2025.108378. PubMed PMID:40562191 .
  18. Gholamalizadeh, H, Baradaran Rahimi, V, Askari, VR. Orphan G Protein-Coupled Receptors: A Novel Research Frontier in Autism and Associated Disorders. Arch Pharm (Weinheim). 2025;358 (6):e70018. doi: 10.1002/ardp.70018. PubMed PMID:40538218 .
  19. Sharma, M, Grover, M, Sharma, N, Shukla, VK, Suryavanshi, SJ. Comparative Review of Clinical Trial Regulations in Different Countries: Current Scenario and Future Prospect. Rev Recent Clin Trials. 2025; :. doi: 10.2174/0115748871365320250529125930. PubMed PMID:40511823 .
  20. Wang, J, Li, H, Liu, H. RD-LIVES: A Living Evidence Synthesis System for Rare Disease Treatment Efficacy and Safety. AMIA Jt Summits Transl Sci Proc. 2025;2025 :607-613. . PubMed PMID:40502246 PubMed Central PMC12150729.
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