PUBLICATIONS

Publications on Orphan Drugs

  1. Khan, MS, Borlaug, BA, Butler, J, Gomberg-Maitland, M, Humbert, M, Lam, CSP et al.. Pulmonary Hypertension Associated With Left Heart Disease: Challenges, Emerging Strategies, and Future Directions. J Am Coll Cardiol. 2026; :. doi: 10.1016/j.jacc.2026.01.024. PubMed PMID:41811276 .
  2. Kayki-Mutlu, G, Aksoyalp, ZS, Wojnowski, L, Michel, MC. A year in pharmacology: new drugs approved by the US food and drug administration in 2025. Naunyn Schmiedebergs Arch Pharmacol. 2026; :. doi: 10.1007/s00210-026-05193-0. PubMed PMID:41807809 .
  3. Manders, E, Keestra, S, Bannenberg, W, van der Wel, V, de Visser, S, Hollak, C et al.. A cost-based-plus pricing approach for repurposed tiratricol in the treatment of Allan-Herndon-Dudley syndrome. Orphanet J Rare Dis. 2026; :. doi: 10.1186/s13023-026-04269-7. PubMed PMID:41787529 .
  4. Papapetropoulos, A, Topouzis, S, Alexander, SPH, Cortese-Krott, MM, Helyes, Z, Martemyanov, K et al.. Novel drugs approved by the EMA, the FDA and the MHRA in 2025: A year in review. Br J Pharmacol. 2026; :. doi: 10.1111/bph.70376. PubMed PMID:41771767 .
  5. Baynam, G, Boycott, KM, Johnson, K, Mckay, L. Language in rare disease: a call for systemic and empathetic action. Lancet. 2026; :. doi: 10.1016/S0140-6736(26)00359-4. PubMed PMID:41765026 .
  6. Miri, M, Abuloha, S, Niu, S, Alshehri, A, Harvey, BP, Svensson, M et al.. Do U.S. cancer drug sales reflect clinical benefit? Evidence from solid tumor drugs from 2008 to 2022. J Cancer Policy. 2026;48 :100723. doi: 10.1016/j.jcpo.2026.100723. PubMed PMID:41763649 .
  7. Jeon, CW, Yoon, JH, Kim, JE. QbD-Based Formulation Development of Amiodarone Hydrochloride Tablet. Pharmaceutics. 2026;18 (2):. doi: 10.3390/pharmaceutics18020264. PubMed PMID:41755005 PubMed Central PMC12944620.
  8. Dorgó, AM, Gunkl-Tóth, L, Nagy, G. Pathogenic Drivers of Difficult-to-Treat Rheumatoid Arthritis: Synovium and Beyond. Int J Mol Sci. 2026;27 (4):. doi: 10.3390/ijms27041860. PubMed PMID:41751996 PubMed Central PMC12940380.
  9. Afonso, S, Gonçalves, J, Brinca, AT, Rosendo, LM, Rosado, T, Duarte, AP et al.. Cannabinoid Therapies in Less-Common Disorders: Clinical Evidence and Formulation Strategies. Diseases. 2026;14 (2):. doi: 10.3390/diseases14020083. PubMed PMID:41745121 PubMed Central PMC12939546.
  10. Piccaluga, PP, Cimmino, L, Tsekhovska, V, Cimatti, P, Innocenti, C, Seidenari, S et al.. From Cell Lines to Avatars: Charting the Future of Preclinical Modeling in T-Cell Malignancies. Cells. 2026;15 (4):. doi: 10.3390/cells15040368. PubMed PMID:41744811 PubMed Central PMC12939640.
  11. Li, Y, Liu, Z, Huang, R, Liu, Y. The imperative for national legislation on rare diseases in China: A policy review and call to action. Intractable Rare Dis Res. 2026;15 (1):4-10. doi: 10.5582/irdr.2025.01058. PubMed PMID:41744018 PubMed Central PMC12932008.
  12. Tang, W. From system building to global engagement: Current significance of and future directions for rare disease research in China. Intractable Rare Dis Res. 2026;15 (1):1-3. doi: 10.5582/irdr.2026.01015. PubMed PMID:41744017 PubMed Central PMC12932007.
  13. Hu, S. Progress in the research and pharmacoeconomic evaluation of drugs and devices for rare diseases in China. Intractable Rare Dis Res. 2026;15 (1):11-16. doi: 10.5582/irdr.2025.01071. PubMed PMID:41744015 PubMed Central PMC12932010.
  14. Rosenberg, N, Hollak, CEM, Haverhoek, VA, Stoyanova-Beninska, V, Pasmooij, AMG, De Visser, SJ et al.. Tracing the path of non-industry-driven medicine development for rare diseases through regulatory interactions at the European Medicines Agency. Drug Discov Today. 2026;31 (2):104631. doi: 10.1016/j.drudis.2026.104631. PubMed PMID:41722896 .
  15. Fletcher, EP, Kim, C, Mei, H, AbuAsal, B, Samuels, S, Madabushi, R et al.. Informing Dose for Pediatric Rare Diseases-A Survey of Recent Orphan Drugs Approvals. Clin Transl Sci. 2026;19 (2):e70503. doi: 10.1111/cts.70503. PubMed PMID:41707029 PubMed Central PMC12916015.
  16. Marcellusi, A, Maggioni, AP, Mandorino, AL, Caprari, F, Marzona, I, Jommi, C et al.. [Not Available]. Glob Reg Health Technol Assess. 2026;13 :49-54. doi: 10.33393/grhta.2026.3604. PubMed PMID:41705260 PubMed Central PMC12908649.
  17. Hollak, CEM, Rosenberg, N, Post, C, Stolwijk, N, Manders, E, Schoenmakers, D et al.. Medicine Development and Access for Rare Diseases: Can We Do Better?. J Inherit Metab Dis. 2026;49 (2):e70146. doi: 10.1002/jimd.70146. PubMed PMID:41702420 PubMed Central PMC12912821.
  18. Jiao, Y, Yuan, F, Liu, T, Han, J, Sun, J, Chu, S et al.. Patent Analysis Perspectives on China's Orphan Drug R&D: Status and Technology Characteristics. Ther Innov Regul Sci. 2026; :. doi: 10.1007/s43441-026-00927-x. PubMed PMID:41697637 .
  19. van der Zanden, TM, Mooij, MG, de Hoog, M, de Wildt, SN. Beyond the label: Rethinking off-label drug use in paediatrics. Towards a scientifically grounded and safer future for paediatric pharmacotherapy. Br J Clin Pharmacol. 2026; :. doi: 10.1002/bcp.70461. PubMed PMID:41693205 .
  20. Nakai, K. Japan's Pharmaceutical Regulatory Reform to Overcome Drug Loss Issue. Ther Innov Regul Sci. 2026; :. doi: 10.1007/s43441-026-00928-w. PubMed PMID:41691137 .
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