PUBLICATIONS

Publications on Orphan Drugs

  1. Zheng, ZZ, Chen, L, Hu, GS, Peng, BL, Huang, QX, Wang, A et al.. Nuclear receptor ESRRA promotes ERα-positive breast cancer through dual action on super enhancers and promoters to regulate gene transcriptional programs. Sci Bull (Beijing). 2025; :. doi: 10.1016/j.scib.2025.09.044. PubMed PMID:41111053 .
  2. Herta, T, Dietz-Fricke, C, Demir, M, Sprinzl, K, Pfeiffer, KH, Grottenthaler, JM et al.. Impact of Handling Perception and Language Barriers on Virologic Response to Daily Subcutaneous Bulevirtide in Hepatitis D. Liver Int. 2025;45 (11):e70404. doi: 10.1111/liv.70404. PubMed PMID:41108575 PubMed Central PMC12535276.
  3. Alipourgivi, F, Lu, T. Celebrating the 40-Year Milestone: NF-ĸB in Oncoimmunity. Cancer Lett. 2025; :218087. doi: 10.1016/j.canlet.2025.218087. PubMed PMID:41106769 .
  4. GBD 2023 Causes of Death Collaborators. Global burden of 292 causes of death in 204 countries and territories and 660 subnational locations, 1990-2023: a systematic analysis for the Global Burden of Disease Study 2023. Lancet. 2025;406 (10513):1811-1872. doi: 10.1016/S0140-6736(25)01917-8. PubMed PMID:41092928 .
  5. GBD 2023 Disease and Injury and Risk Factor Collaborators. Burden of 375 diseases and injuries, risk-attributable burden of 88 risk factors, and healthy life expectancy in 204 countries and territories, including 660 subnational locations, 1990-2023: a systematic analysis for the Global Burden of Disease Study 2023. Lancet. 2025;406 (10513):1873-1922. doi: 10.1016/S0140-6736(25)01637-X. PubMed PMID:41092926 .
  6. Boucheham, A, Franco, JM, Bär, S, MacDonald, E, Zuttion, S, Blagec, L et al.. Galectin-3 Mediated Endocytosis of the Orphan G-Protein-Coupled Receptor GPRC5A. Cells. 2025;14 (19):. doi: 10.3390/cells14191571. PubMed PMID:41090797 PubMed Central PMC12524283.
  7. Godoy, CB, Ramachandran, R, Sapre, P, Ross, JS. Confirmatory evidence supporting single pivotal trial new drug approvals by the Food and Drug Administration, 2015 through 2023. Clin Trials. 2025; :17407745251376620. doi: 10.1177/17407745251376620. PubMed PMID:41090443 .
  8. Hopefl, R, Wang, J, Mohan, AR, Sun, WJ, Kim, MJ, Hu, M et al.. Predictive Analysis for First Submission of Generic Drug Application for Orphan Drug Products Using Random Survival Forest. Clin Transl Sci. 2025;18 (10):e70365. doi: 10.1111/cts.70365. PubMed PMID:41046470 PubMed Central PMC12497357.
  9. Chinoy, J, Meller, C, de Wet, H. An Emerging Paradigm for ABCC5/MRP5 Function in Human Physiology. Int J Mol Sci. 2025;26 (18):. doi: 10.3390/ijms26189211. PubMed PMID:41009771 PubMed Central PMC12470876.
  10. Akodad, S, De Smedt, D, Stevens, H. Societal participation in the development of orphan drugs: a systematic review. Front Med (Lausanne). 2025;12 :1653304. doi: 10.3389/fmed.2025.1653304. PubMed PMID:41001376 PubMed Central PMC12457289.
  11. Goh, M, McEnany, M, Freeman, R, Newton, M, Kesselheim, AS, Outterson, K et al.. Bridging the fair share gap for antibacterial innovation: an observational analysis of antibacterial revenues in the G7 and EU27. EClinicalMedicine. 2025;88 :103485. doi: 10.1016/j.eclinm.2025.103485. PubMed PMID:40989242 PubMed Central PMC12452676.
  12. Price, ML, Wyatt, RA, Crastin, A, Jamaluddin, A, Hardy, RS, Frost, M et al.. G protein-coupled receptor 35 stimulation reduces osteoclast activity in primary human bone cells. JBMR Plus. 2025;9 (10):ziaf131. doi: 10.1093/jbmrpl/ziaf131. PubMed PMID:40978132 PubMed Central PMC12445851.
  13. Lee, SH, Lee, SK, Lee, JH, Bang, JS. Determinants of orphan drug health technology assessment in South Korea: an empirical analysis. Front Pharmacol. 2025;16 :1619984. doi: 10.3389/fphar.2025.1619984. PubMed PMID:40963687 PubMed Central PMC12436107.
  14. Villano, G, D'Onofrio, G, Riva, A, Santangelo, A, Rose, K, Striano, P et al.. Navigating regulatory challenges in pediatric epilepsy pharmacotherapy: pathways to safe and effective treatments. Expert Opin Pharmacother. 2025;26 (13):1399-1414. doi: 10.1080/14656566.2025.2563279. PubMed PMID:40960486 .
  15. Mo, Q, He, J, Guo, Q, Yang, Y. Drug lag and associated factors of orphan drugs approved by the U.S. in China. Front Pharmacol. 2025;16 :1595497. doi: 10.3389/fphar.2025.1595497. PubMed PMID:40949125 PubMed Central PMC12425770.
  16. Dieu, DC, Pernice, HF, Muratovic, H, Wetzel, PJ, Barzen, G, Wieder, NW et al.. Real-world treatment management in hereditary transthyretin amyloidosis - an experience report and proposal for therapy switch decision criteria. Neurol Res Pract. 2025;7 (1):65. doi: 10.1186/s42466-025-00428-6. PubMed PMID:40940654 PubMed Central PMC12432994.
  17. Valinejad, J, Xu, Y, Zhu, Q. An application of studying FAERS data to Enhance Drug Safety and Treatment Outcomes in Rare Diseases. Proceedings (IEEE Int Conf Bioinformatics Biomed). 2024;2024 :4575-4578. doi: 10.1109/bibm62325.2024.10822513. PubMed PMID:40933844 PubMed Central PMC12419809.
  18. Basu, A, Thomas, SK, Chapman, RH, Spangler, J. HTA Evidence in Rare Diseases: Just Rare or Also Special?. Pharmacoeconomics. 2025;43 (11):1271-1279. doi: 10.1007/s40273-025-01538-4. PubMed PMID:40924270 PubMed Central PMC12534280.
  19. Papadopoulos, F, Visintin, E, Kyriopoulos, I, Kanavos, P. Why drugs fail health technology assessment: a comparative analysis of health technology assessment rejections across seven OECD countries. Health Econ Policy Law. 2025;20 (3):264-283. doi: 10.1017/S1744133125100212. PubMed PMID:40922553 .
  20. Han, Y, Zhuo, Q, Chen, N, Zhang, H, Lin, L, Song, P et al.. Rare but not to be overlooked: Epidemiology and strategies for rare dermatological diseases in China. Intractable Rare Dis Res. 2025;14 (3):151-161. doi: 10.5582/irdr.2025.01039. PubMed PMID:40904638 PubMed Central PMC12403912.
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