PUBLICATIONS

Publications on Orphan Drugs

  1. GBD 2023 MASLD Collaborators. Global burden of metabolic dysfunction-associated steatotic liver disease, 1990-2023, and projections to 2050: a systematic analysis for the Global Burden of Disease Study 2023. Lancet Gastroenterol Hepatol. 2026; :. doi: 10.1016/S2468-1253(26)00011-7. PubMed PMID:41990758 .
  2. Peffault de Latour, R, Pibre, S, Vercruyssen, J, Desjuzeur-Bellais, ML, Pau, D, Bénard, S et al.. Epidemiology and care management of Paroxysmal Nocturnal Hemoglobinuria (PNH) in a real-world setting in France: Description from the French National Hospitalization Database. PLoS One. 2026;21 (4):e0339715. doi: 10.1371/journal.pone.0339715. PubMed PMID:41990028 PubMed Central PMC13086315.
  3. Curtis, D, Zhao, X, Owen, NM, Azamian, MS, Lalani, SR, Keller, K et al.. New genotype-phenotype correlations and management recommendations for individuals with RERE variants. Genet Med. 2026; :102580. doi: 10.1016/j.gim.2026.102580. PubMed PMID:41988794 .
  4. Bamgboye, AO, Coles, LD, Suriyapakorn, B, Mishra, U, Kriel, RL, Leppik, IE et al.. Population Pharmacokinetic Modeling of Intravenous Topiramate in Patients with Epilepsy or Migraine. J Clin Pharmacol. 2026;66 (4):e70191. doi: 10.1002/jcph.70191. PubMed PMID:41987705 PubMed Central PMC13084296.
  5. Mori, T, Hirano, KI, Miyauchi, H, Ikeda, Y, Higashi, M, Nakano, Y et al.. Triglyceride deposit cardiomyovasculopathy: A new class of cardiovascular disease. J Cardiol. 2026; :. doi: 10.1016/j.jjcc.2026.04.003. PubMed PMID:41980667 .
  6. Vesković, M, Šutulović, N, Djuric, E, Hrnčić, D, Marković, AR, Stanojlović, O et al.. Vitamin D as a Regulator of the Biological Clock-Implications for Circadian-Metabolic Dysregulation. Int J Mol Sci. 2026;27 (7):. doi: 10.3390/ijms27073243. PubMed PMID:41977424 PubMed Central PMC13074111.
  7. Ruiz, AA, Shinabargar, E, Slovenski, I, Large, K, Vieira, M, Moon, S et al.. Increasing access to CAR-T therapy: a case study of an academic hospital's alternative innovation model. Orphanet J Rare Dis. 2026; :. doi: 10.1186/s13023-026-04320-7. PubMed PMID:41964091 .
  8. 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 .
  9. 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.
  10. 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 .
  11. 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 .
  12. 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.
  13. 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 .
  14. 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.
  15. 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 .
  16. 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.
  17. 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.
  18. 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 .
  19. 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 .
  20. 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 .
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