PUBLICATIONS

Publications on Orphan Drugs

  1. Mukuku, O, Govender, K, Wembonyama, SO, Kiakuvue, YN. Magnitude and characteristics of unsuppressed HIV viral load in children and adolescents on antiretroviral therapy in sub-Saharan Africa: a systematic review and meta-analysis. Lancet HIV. 2025; :. doi: 10.1016/S2352-3018(25)00039-6. PubMed PMID:40473447 .
  2. Goyal, PK, Sharma, V, Sangwan, K. Pharmacological Insights on USFDA-Approved Novel Drug Therapies in the Year 2023. Curr Drug Discov Technol. 2025; :. doi: 10.2174/0115701638379447250526075127. PubMed PMID:40464188 .
  3. Klok, FA, Barco, S, Bertoletti, L, Bhalla, S, Dubois, S, Le Gal, G et al.. Optimal approach to performing and reporting computed tomography angiography for suspected acute pulmonary embolism: a clinical consensus statement of the ESC Working Group on Pulmonary Circulation & Right Ventricular Function, the Fleischner Society, the Association for Acute CardioVascular Care (ACVC) and the European Association of Cardiovascular Imaging (EACVI) of the ESC, endorsed by European Respiratory Society (ERS), Asian Society of Thoracic Radiology (ASTR), European Society of Thoracic Imaging (ESTI), and Society of Thoracic Radiology (STR). Eur Heart J Cardiovasc Imaging. 2025; :. doi: 10.1093/ehjci/jeaf050. PubMed PMID:40457988 .
  4. Hahl, E, Kurko, T, Koskinen, H, Airaksinen, M, Sarnola, K. EMA approved orphan medicines since the implementation of the orphan legislation. Orphanet J Rare Dis. 2025;20 (1):266. doi: 10.1186/s13023-025-03756-7. PubMed PMID:40457478 PubMed Central PMC12131831.
  5. Suwattanapreeda, S, Hirunrassamee, S, Sooksriwong, C, Maluangnon, K, Chuachantra, T, Kuchaisit, K et al.. Unlocking access: a comprehensive analysis of medicines accessibility for rare diseases in Thailand. Orphanet J Rare Dis. 2025;20 (1):258. doi: 10.1186/s13023-025-03754-9. PubMed PMID:40437602 PubMed Central PMC12121248.
  6. Wen, X, Jin, G, Wu, C. Visual Research of Global Orphan Drug from a Bibliometric Perspective. Drug Des Devel Ther. 2025;19 :4201-4220. doi: 10.2147/DDDT.S506112. PubMed PMID:40416799 PubMed Central PMC12103199.
  7. Fanciulli, A, Caliò, B, Schmidt, S, Goebel, G, Leys, F, Radl, K et al.. MeDeMSA care study protocol: developing personalized best medical care with integrated mobile palliative and telemedicine support for individuals with multiple system atrophy. J Neural Transm (Vienna). 2025; :. doi: 10.1007/s00702-025-02933-z. PubMed PMID:40411588 .
  8. Hu, R, Liu, D, Sun, X, Yu, Z. New drugs approved by European medicines agency during 2024. Eur J Med Chem. 2025;294 :117761. doi: 10.1016/j.ejmech.2025.117761. PubMed PMID:40409054 .
  9. Ahmad, A, Simran, S, Kalokhe, VM, Musthafa, F, Gangawane, VS, Choudhary, KK et al.. Genetic renaissance: a cross-regional analysis of the approval landscape of authorised gene therapeutics in paediatrics, challenges and future prospects. J Community Genet. 2025; :. doi: 10.1007/s12687-025-00799-y. PubMed PMID:40397378 .
  10. Williamson, J, Hasan, SS, Gc, VS. Economic and developmental impacts of FDA designations: a systematic review and meta-analysis. Expert Rev Pharmacoecon Outcomes Res. 2025; :1-11. doi: 10.1080/14737167.2025.2507426. PubMed PMID:40387898 .
  11. Shanmugaraj, B, Ravi, K, Baladevan, K. Plant molecular farming: a promising frontier for orphan drug production. Biotechnol Lett. 2025;47 (3):56. doi: 10.1007/s10529-025-03596-2. PubMed PMID:40381123 .
  12. Abozaid, GM, Al-Omar, HA, Alrabiah, A, AlMuaither, A, McKnight, AJ. Towards a sustainable rare disease and orphan drug ecosystem in Saudi Arabia: policy insights from a multi-stakeholder workshop. Front Pharmacol. 2025;16 :1583477. doi: 10.3389/fphar.2025.1583477. PubMed PMID:40376269 PubMed Central PMC12078156.
  13. Chippi, C. Amgen Enablement and its Undue Burden on an Overburdened Biomedical Industry. J Law Health. 2025;38 (3):383-401. . PubMed PMID:40338962 .
  14. Zhang, L, Gatlin, V, Gupta, S, Salinas, ML, Romero, S, Cai, JJ et al.. Expression of Prooncogenic Nuclear Receptor 4A (NR4A)-Regulated Genes β1-Integrin and G9a Inhibited by Dual NR4A1/2 Ligands. Int J Mol Sci. 2025;26 (8):. doi: 10.3390/ijms26083909. PubMed PMID:40332813 PubMed Central PMC12028307.
  15. Wang, H, Zhang, G, Zhao, C, Xue, Y, Zhu, D, Chang, Y et al.. RORγt agonist LYC-55716 potentiates IFN-α's efficacy in hepatocellular carcinoma through enhancing cytotoxicity of Tc17 cells and infiltration of CD8+ T cells. Biochem Pharmacol. 2025;238 :116963. doi: 10.1016/j.bcp.2025.116963. PubMed PMID:40312017 .
  16. Shao, Y, Mei, Y, Tan, Y, Yang, M, Wu, H. The regulatory functions of G protein-coupled receptors signaling pathways in B cell differentiation and development contributing to autoimmune diseases. Cell Biosci. 2025;15 (1):57. doi: 10.1186/s13578-025-01398-7. PubMed PMID:40307944 PubMed Central PMC12042570.
  17. Awada, N. Access to drugs for rare diseases (DRD) in Canada: a comprehensive review of the provincial DRD-specific programs. Ther Adv Rare Dis. 2025;6 :26330040251335660. doi: 10.1177/26330040251335660. PubMed PMID:40302781 PubMed Central PMC12038190.
  18. Young, CM, Phares, SE, Kennedy, A, Sullivan, J, McGowan, B, Trusheim, MR et al.. Pediatric-onset rare disease therapy pipeline yields hope for some and gaps for many: 10-year projection of approvals, treated patients, and list price revenues. J Manag Care Spec Pharm. 2025;31 (5):491-498. doi: 10.18553/jmcp.2025.31.5.491. PubMed PMID:40298308 PubMed Central PMC12039501.
  19. Russell, IC, Lee, D, Wootten, D, Sexton, PM, Bumbak, F. Cryoelectron microscopy as a tool for illuminating activation mechanisms of human class A orphan G protein-coupled receptors. Pharmacol Rev. 2025;77 (3):100056. doi: 10.1016/j.pharmr.2025.100056. PubMed PMID:40286430 .
  20. Chen, T, An, Q, Zhang, T, Yan, S, Li, J, Song, X et al.. Mechanism of Penoxsulam's Effect on Chlorophyll Synthesis and the Metabolism of Foxtail Millet. Plants (Basel). 2025;14 (8):. doi: 10.3390/plants14081211. PubMed PMID:40284098 PubMed Central PMC12030371.
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