PUBLICATIONS

Publications on Orphan Drugs

  1. 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 .
  2. 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.
  3. 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 .
  4. 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 .
  5. 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.
  6. 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.
  7. 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.
  8. 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.
  9. 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 .
  10. 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 .
  11. 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.
  12. 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.
  13. 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 .
  14. 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 .
  15. 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 .
  16. 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.
  17. 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 .
  18. 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 .
  19. 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 .
  20. 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 .
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