PUBLICATIONS

Publications on Orphan Drugs

  1. Hong, J, Bae, EY, Cha, S, Lee, J. The value-for-money assessment and funding arrangements for high-priced drugs in an era of uncertainty: a comparative analysis of national health technology assessment agencies in South Korea, England, Australia, and Canada. BMC Health Serv Res. 2025;25 (1):74. doi: 10.1186/s12913-025-12207-9. PubMed PMID:39810177 PubMed Central PMC11731375.
  2. Chen, J, Zhao, T, Hong, W, Li, H, Ao, M, Zhong, Y et al.. Discovery of a novel exceptionally potent and orally active Nur77 ligand NB1 with a distinct binding mode for cancer therapy. Acta Pharm Sin B. 2024;14 (12):5493-5504. doi: 10.1016/j.apsb.2024.07.012. PubMed PMID:39807329 PubMed Central PMC11725030.
  3. Windfuhr, F, Larsson, K, Framke, T, Lasch, F. Which clinical trial designs and statistical approaches have been used in assessments of orphan maintenance by the European Medicines Agency between 2012 and 2022? A cross-sectional study. BMJ Open. 2024;14 (12):e086171. doi: 10.1136/bmjopen-2024-086171. PubMed PMID:39806587 PubMed Central PMC11667437.
  4. Odnoletkova, I, Chalon, PX, Devriese, S, Cleemput, I. Projections of Public Spending on Pharmaceuticals: A Review of Methods. Pharmacoeconomics. 2025; :. doi: 10.1007/s40273-024-01465-w. PubMed PMID:39798038 .
  5. Rose, K, Grant-Kels, JM, Striano, P, Russo, E, Ettienne, EB. Deception and Fraud in the Justification of "Pediatric Drug Development": A Challenge at the Interface of Medicine and Law. J Law Med. 2024;31 (3):635-644. . PubMed PMID:39789690 .
  6. Kunicki, PK, Grymm, MT, Pawiński, T, Szulczyk, D, Waligóra, M, Kopeć, G et al.. A simple HPLC-UV method for monitoring therapeutic adherence in pulmonary arterial hypertension. J Chromatogr B Analyt Technol Biomed Life Sci. 2024;1252 :124443. doi: 10.1016/j.jchromb.2024.124443. PubMed PMID:39787725 .
  7. Alexander, T, Sewerin, P, Strangfeld, A, Schulte, M, Borchert, J, Garcia, TB et al.. Real-World Prevalence, Incidence and Management of Systemic Lupus Erythematosus in Germany: A Retrospective Claims Data Analysis. Rheumatol Ther. 2025; :. doi: 10.1007/s40744-024-00735-5. PubMed PMID:39776056 .
  8. Novick, D. IL-18 and IL-18BP: A Unique Dyad in Health and Disease. Int J Mol Sci. 2024;25 (24):. doi: 10.3390/ijms252413505. PubMed PMID:39769266 PubMed Central PMC11727785.
  9. Gandjour, A. Reimbursement prices of new, innovative medicines in Germany: a comparison of negotiation and cost-effectiveness analysis. Health Econ Policy Law. 2025; :1-12. doi: 10.1017/S1744133124000288. PubMed PMID:39763240 .
  10. JPMA MIDD Task force, Sasaki, T, Katsube, T, Hayato, S, Yamaguchi, S, Tanaka, J et al.. Application of model-informed drug development (MIDD) for dose selection in regulatory submissions for drug approval in Japan. J Pharmacokinet Pharmacodyn. 2025;52 (1):10. doi: 10.1007/s10928-024-09954-3. PubMed PMID:39762622 .
  11. Lai, X, Chen, Y, Huang, R, Zhu, F, Huang, L, Chen, N et al.. Lentinan inhibits melanoma development by regulating the AKT/Nur77/Bcl-2 signaling axis. J Cancer. 2025;16 (2):543-557. doi: 10.7150/jca.100863. PubMed PMID:39744474 PubMed Central PMC11685690.
  12. Delgado-Nungaray, JA, Figueroa-Yáñez, LJ, Reynaga-Delgado, E, Corona-España, AM, Gonzalez-Reynoso, O. Unveiling the endogenous CRISPR-Cas system in Pseudomonas aeruginosa PAO1. PLoS One. 2024;19 (12):e0312783. doi: 10.1371/journal.pone.0312783. PubMed PMID:39739718 PubMed Central PMC11687729.
  13. Yin, L, Fu, Z, Wang, M, Liu, B, Sun, X, Liu, K et al.. A prodrug nanodevice co-delivering docetaxel and ROR1 siRNA for enhanced triple negative breast cancer therapy. Acta Biomater. 2024; :. doi: 10.1016/j.actbio.2024.12.055. PubMed PMID:39730101 .
  14. Rosenberg, N, Manders, E, van den Berg, S, Deesker, LJ, Garrelfs, SF, de Visser, SJ et al.. Application of four pricing models for orphan medicines: a case study for lumasiran. Orphanet J Rare Dis. 2024;19 (1):485. doi: 10.1186/s13023-024-03446-w. PubMed PMID:39716306 PubMed Central PMC11667848.
  15. Gangwal, A, Lavecchia, A. AI-Driven Drug Discovery for Rare Diseases. J Chem Inf Model. 2024; :. doi: 10.1021/acs.jcim.4c01966. PubMed PMID:39689164 .
  16. Gensapa, R, Pandey, V, Saha, SK, Mukherjee, SK, Jha, A. Inter-relational dynamics of factors affecting the emergence of orphan drugs. Ann Pharm Fr. 2024; :. doi: 10.1016/j.pharma.2024.12.005. PubMed PMID:39675421 .
  17. Tomsen-Melero, J, Moltó-Abad, M, Merlo-Mas, J, Díaz-Riascos, ZV, Cristóbal-Lecina, E, Soldevila, A et al.. Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease. Sci Adv. 2024;10 (50):eadq4738. doi: 10.1126/sciadv.adq4738. PubMed PMID:39671483 .
  18. Global Nutrition Target Collaborators. Global, regional, and national progress towards the 2030 global nutrition targets and forecasts to 2050: a systematic analysis for the Global Burden of Disease Study 2021. Lancet. 2025;404 (10471):2543-2583. doi: 10.1016/S0140-6736(24)01821-X. PubMed PMID:39667386 PubMed Central PMC11703702.
  19. Pizzamiglio, C, Stefanetti, RJ, McFarland, R, Thomas, N, Ransley, G, Hugerth, M et al.. Optimizing rare disorder trials: a phase 1a/1b randomized study of KL1333 in adults with mitochondrial disease. Brain. 2025;148 (1):39-46. doi: 10.1093/brain/awae308. PubMed PMID:39657714 PubMed Central PMC11706290.
  20. Kakkilaya, A, Shahzad, M, Bourgeois, FT. FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023. JAMA Pediatr. 2024; :. doi: 10.1001/jamapediatrics.2024.5280. PubMed PMID:39652344 .
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