PUBLICATIONS

Publications on Orphan Drugs

  1. Sharma, M, Grover, M, Sharma, N, Shukla, VK, Suryavanshi, SJ. Comparative Review of Clinical Trial Regulations in Different Countries: Current Scenario and Future Prospect. Rev Recent Clin Trials. 2025; :. doi: 10.2174/0115748871365320250529125930. PubMed PMID:40511823 .
  2. Wang, J, Li, H, Liu, H. RD-LIVES: A Living Evidence Synthesis System for Rare Disease Treatment Efficacy and Safety. AMIA Jt Summits Transl Sci Proc. 2025;2025 :607-613. . PubMed PMID:40502246 PubMed Central PMC12150729.
  3. Lang, IM, Brenot, P, Bouvaist, H, Fadel, E, Jaïs, X, Madani, MM et al.. Balloon Pulmonary Angioplasty for Chronic Thromboembolic Pulmonary Hypertension: Results of an International Multicenter Prospective Registry. J Am Coll Cardiol. 2025;85 (23):2270-2284. doi: 10.1016/j.jacc.2025.04.021. PubMed PMID:40499982 .
  4. Romagnoli, A, Marcellusi, A, Lasala, R. [Analysis of the reimbursement times of antitumor drugs in Italy in relation to the clinical benefit expressed by the European Society for Medical Oncology (ESMO-MCBS).]. Recenti Prog Med. 2025;116 (6):371-379. doi: 10.1701/4518.45175. PubMed PMID:40491402 .
  5. Subramanian, N, Subramanian, A. Determinants of cancer drug prices in Germany and France: a multiple regression analysis. Expert Rev Pharmacoecon Outcomes Res. 2025; :1-8. doi: 10.1080/14737167.2025.2518140. PubMed PMID:40481741 .
  6. Climente Martí, M, Edo Solsona, MD, Abad Sazatornil, MR, Bachiller Cacho, MP, Fernández Espinola, S, Guijarro Martínez, P et al.. [Translated article] OrPhar-SEFH 2024-2027 Strategic Plan. Farm Hosp. 2025; :. doi: 10.1016/j.farma.2025.05.004. PubMed PMID:40480898 .
  7. 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 .
  8. 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 .
  9. 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 .
  10. 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.
  11. 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.
  12. 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.
  13. 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 .
  14. 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 .
  15. 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 .
  16. 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 .
  17. 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 .
  18. 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.
  19. Chippi, C. Amgen Enablement and its Undue Burden on an Overburdened Biomedical Industry. J Law Health. 2025;38 (3):383-401. . PubMed PMID:40338962 .
  20. 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.
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