PUBLICATIONS

Publications on Orphan Drugs

  1. Kostadinov, K, Popova-Sotirova, I, Marinova, Y, Musurlieva, N, Iskrov, G, Stefanov, R et al.. Availability and Access to Orphan Drugs for Rare Cancers in Bulgaria: Analysis of Delays and Public Expenditures. Cancers (Basel). 2024;16 (8):. doi: 10.3390/cancers16081489. PubMed PMID:38672571 PubMed Central PMC11048562.
  2. Risse, J, Krzemien, M, Schnalke, J, Heinemann, T. Towards ethical drug pricing: the European Orphan Genomic Therapies Fund. Gene Ther. 2024; :. doi: 10.1038/s41434-024-00452-2. PubMed PMID:38658672 .
  3. Qiu, Y, Hou, Y, Gohel, D, Zhou, Y, Xu, J, Bykova, M et al.. Systematic characterization of multi-omics landscape between gut microbial metabolites and GPCRome in Alzheimer's disease. Cell Rep. 2024;43 (5):114128. doi: 10.1016/j.celrep.2024.114128. PubMed PMID:38652661 .
  4. Flynn, MB, Flynn, JF, Palacios, AM. Capitalizing on Hope: Questionable Marketing Approval and Pricing of a New ALS Drug. Int J Soc Determinants Health Health Serv. 2024; :27551938241247778. doi: 10.1177/27551938241247778. PubMed PMID:38646691 .
  5. Stević, I, Janković, SM, Georgiev, AM, Marinković, V, Lakić, D. Factors associated with hematological adverse reactions of drugs authorized via the centralized procedure. Sci Rep. 2024;14 (1):9074. doi: 10.1038/s41598-024-59710-3. PubMed PMID:38643204 PubMed Central PMC11032331.
  6. Zhang, H, Diwu, YC, Zhao, EL, Duan, LQ, Han, XY, Wang, DK et al.. [Xixin Decoction regulates Th17/Treg balance and transcription factor expression in senescence-accelerated mouse-prone]. Zhongguo Zhong Yao Za Zhi. 2024;49 (5):1353-1360. doi: 10.19540/j.cnki.cjcmm.20231210.502. PubMed PMID:38621983 .
  7. Wang, DE, Hassanein, M, Razvi, Y, Shaul, RZ, Denburg, A. Institutional Priority-Setting for Novel Drugs and Therapeutics: A Qualitative Systematic Review. Int J Health Policy Manag. 2024;13 :7494. doi: 10.34172/ijhpm.2024.7494. PubMed PMID:38618836 PubMed Central PMC11016276.
  8. Grand, TS, Ren, S, Hall, J, Åström, DO, Regnier, S, Thokala, P et al.. Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review. Pharmacoeconomics. 2024; :. doi: 10.1007/s40273-024-01370-2. PubMed PMID:38616217 .
  9. Juan, C, Gallo, L, Gonzalez Vidal, N. Development of Losartan Orally Disintegrating Tablets by Direct Compression: a Cost-Effective Approach to Improve Paediatric Patient's Compliance. AAPS PharmSciTech. 2024;25 (4):79. doi: 10.1208/s12249-024-02796-9. PubMed PMID:38589718 .
  10. GBD 2021 Causes of Death Collaborators. Global burden of 288 causes of death and life expectancy decomposition in 204 countries and territories and 811 subnational locations, 1990-2021: a systematic analysis for the Global Burden of Disease Study 2021. Lancet. 2024; :. doi: 10.1016/S0140-6736(24)00367-2. PubMed PMID:38582094 .
  11. Schouest, B, Bindal, KR. Diversity Plans and Postmarketing Studies: First Impressions of Anticipated Diversity Requirements in the United States. Ther Innov Regul Sci. 2024; :. doi: 10.1007/s43441-024-00643-4. PubMed PMID:38568346 .
  12. Alnaqbi, KA, Elezbawy, B, Fasseeh, AN, Bangash, AR, Elshamy, A, Shendi, H et al.. Development of the Emirates Multi-Criteria Decision Analysis Tool for Orphan Drugs. Cureus. 2024;16 (2):e55215. doi: 10.7759/cureus.55215. PubMed PMID:38558740 PubMed Central PMC10981202.
  13. Kinch, MS, Kraft, Z, Schwartz, T. 2023 in review: FDA approvals of new medicines. Drug Discov Today. 2024;29 (5):103966. doi: 10.1016/j.drudis.2024.103966. PubMed PMID:38552777 .
  14. Ruggiero, R, Balzano, N, Nicoletti, MM, di Mauro, G, Fraenza, F, Campitiello, MR et al.. Real-World Safety Data of the Orphan Drug Onasemnogene Abeparvovec (Zolgensma®) for the SMA Rare Disease: A Pharmacovigilance Study Based on the EMA Adverse Event Reporting System. Pharmaceuticals (Basel). 2024;17 (3):. doi: 10.3390/ph17030394. PubMed PMID:38543180 PubMed Central PMC10974574.
  15. Jakubowski, S, Kawalec, P, Holko, P, Kowalska-Bobko, I, Kamusheva, M, Petrova, G et al.. Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries. Front Pharmacol. 2024;15 :1369178. doi: 10.3389/fphar.2024.1369178. PubMed PMID:38523639 PubMed Central PMC10957562.
  16. Liu, C, Liu, Y, Ou, L, Qi, Y, Zhang, J. Recent landscape and trends for industry-sponsored pediatric clinical trials in China from 2013 to 2022. Pediatr Investig. 2024;8 (1):12-20. doi: 10.1002/ped4.12409. PubMed PMID:38516137 PubMed Central PMC10951485.
  17. Pandina, GJ, Busner, J, Kempf, L, Fallon, J, Alphs, LD, Acosta, MT et al.. Ensuring Stakeholder Feedback in the Design and Conduct of Clinical Trials for Rare Diseases: ISCTM Position Paper of the Orphan Disease Working Group. Innov Clin Neurosci. 2024;21 (1-3):52-60. . PubMed PMID:38495603 PubMed Central PMC10941866.
  18. Chen, X, Gao, M, Xia, Y, Wang, X, Qin, J, He, H et al.. Phase separation of Nur77 mediates XS561-induced apoptosis by promoting the formation of Nur77/Bcl-2 condensates. Acta Pharm Sin B. 2024;14 (3):1204-1221. doi: 10.1016/j.apsb.2023.11.017. PubMed PMID:38486987 PubMed Central PMC10935061.
  19. Man, A, Groeneweg, GSS, Ross, CJD, Carleton, BC. The Role of Pharmacogenomics in Rare Diseases. Drug Saf. 2024; :. doi: 10.1007/s40264-024-01416-6. PubMed PMID:38483768 .
  20. Kalland, ME, Pose-Boirazian, T, Palomo, GM, Naumann-Winter, F, Costa, E, Matusevicius, D et al.. Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation. Gene Ther. 2024; :. doi: 10.1038/s41434-024-00446-0. PubMed PMID:38480914 .
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