PUBLICATIONS

Publications on Orphan Drugs

  1. Siebert, M, Caquelin, L, Naudet, F, Ross, JS, Ramachandran, R. Evidence supporting European Medicines Agency drug approvals (2020-2023): A cross-sectional study of trial design and outcomes. Clin Trials. 2026; :17407745261437363. doi: 10.1177/17407745261437363. PubMed PMID:41944409 .
  2. Melinder, M, Windfuhr, F, Dahlqvist, T, Torre, C, Sepodes, B, Mol, PGM et al.. A Cross-Sectional Survey of Evidence Needs for Medicinal Products in Europe With a Focus on Real-World Evidence. Pharmacoepidemiol Drug Saf. 2026;35 (4):e70358. doi: 10.1002/pds.70358. PubMed PMID:41934228 PubMed Central PMC13049559.
  3. Hashmi, SK, Jamil, MA, de la Fuente, J, Mahlangu, J, Ozelo, MC, Walters, MC et al.. Futuristic global cell and gene therapy regulations based on a review of today's leading frameworks. Cytotherapy. 2026;28 (6):102064. doi: 10.1016/j.jcyt.2026.102064. PubMed PMID:41930807 .
  4. Johnson, EA. Manufacture of the first FDA-approved batches of 900 kilodalton crystalline type a botulinum toxin for clinical use: a personal remembrance. J Neural Transm (Vienna). 2026; :. doi: 10.1007/s00702-026-03124-0. PubMed PMID:41925782 .
  5. He, J, Wei, H, Mo, Q, Liu, Y, Li, X, Yang, Y et al.. A comparative analysis of health technology assessment decision-making for cancer drugs in Germany and England. Front Public Health. 2026;14 :1733239. doi: 10.3389/fpubh.2026.1733239. PubMed PMID:41923768 PubMed Central PMC13035802.
  6. GBD 2023 IHD & Dietary Risk Factors Collaborators. Global, regional and national burden of ischemic heart disease attributable to suboptimal diet, 1990-2023: a Global Burden of Disease study. Nat Med. 2026; :. doi: 10.1038/s41591-026-04250-8. PubMed PMID:41912805 .
  7. Hailemariam, A, Upadhyay, S, Oany, AR, Tsui, WNT, Srivastava, V, Sivaram, G et al.. Brewed Coffee and Its Components Act Through Orphan Nuclear Receptor 4A1 (NR4A1). Nutrients. 2026;18 (6):. doi: 10.3390/nu18060877. PubMed PMID:41901052 PubMed Central PMC13029766.
  8. Roesch, EW, Colpani, V, Dória, GAA, de Oliveira, KHD, Oliveira, C, Peixoto, HM et al.. Cost-Utility Analysis of the Treatment With Ataluren Plus Standard of Care Compared With Standard of Care Alone in Patients With Duchenne Muscular Dystrophy in Brazil. Value Health Reg Issues. 2026; :101623. doi: 10.1016/j.vhri.2026.101623. PubMed PMID:41885666 .
  9. McAllister-Williams, RH, Alva, G, Banov, M, Conway, CR, Cusin, C, De Beer, JC et al.. The Use of Vagus Nerve Stimulation (VNS) in the Management of Patients with Difficult-to-Treat Major Depressive Disorder (MDD): An Expert Consensus Statement. Neuropsychiatr Dis Treat. 2026;22 :554007. doi: 10.2147/NDT.S554007. PubMed PMID:41884079 PubMed Central PMC13008595.
  10. Guglielmelli, P, Breccia, M, Mendicino, F, Martelli, M, Di Renzo, N, Palumbo, GA et al.. Clinical Outcomes of Ruxolitinib Treatment in Patients With IPSS Intermediate-1-Risk Myelofibrosis: Interim Analysis From an Italian, Prospective Study (ROMEI). Hematol Oncol. 2026;44 (2):e70178. doi: 10.1002/hon.70178. PubMed PMID:41877599 PubMed Central PMC13014119.
  11. Beninger, P. Menkes Disease: Another Example of Progress in the Development of Pharmaceuticals for Rare Childhood Diseases, But Much Still Remains to Be Done. Clin Ther. 2026; :. doi: 10.1016/j.clinthera.2026.02.014. PubMed PMID:41876279 .
  12. Yu, YC, Huang, N, Zhao, ZH, Bao, RF, Xu, F, Xu, ZQ et al.. Fluorescence polarization-based discovery of natural RORγ orthosteric inhibitors. Eur J Med Chem. 2026;309 :118778. doi: 10.1016/j.ejmech.2026.118778. PubMed PMID:41849945 .
  13. Heling, LWHJ, Kovač, K, de Vries, CJM, Mashaghi, A. Modulation of Nur77-DNA interactions by the glucocorticoid receptor. Biochem J. 2026;483 (4):429-440. doi: 10.1042/BCJ20250293. PubMed PMID:41847979 .
  14. Rajasekharan, S, Barragan Torres, VA, Pinheiro Gomes, YC, Wilken, L, Overhoff, K, Lin, YC et al.. A genus-wide interaction atlas across NS4B orthologues identifies a conserved role for UFMylation in orthoflavivirus replication. Nat Commun. 2026;17 (1):. doi: 10.1038/s41467-026-70437-9. PubMed PMID:41826287 PubMed Central PMC12992610.
  15. 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 .
  16. 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 .
  17. 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 .
  18. 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 .
  19. Baynam, G, Boycott, KM, Johnson, K, Mckay, L. Language in rare disease: a call for systemic and empathetic action. Lancet. 2026;407 (10535):1216-1218. doi: 10.1016/S0140-6736(26)00359-4. PubMed PMID:41765026 .
  20. 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 .
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