PUBLICATIONS

Publications on Orphan Drugs

  1. Andersen, DB, Albertsen, AB, Wadmann, S, Bech, M. Size Does Matter! Prioritizing Rare Diseases for Luck Egalitarian Reasons. Bioethics. 2026; :. doi: 10.1111/bioe.70136. PubMed PMID:42250269 .
  2. Xie, G, Wang, Q, Du, M, Zhou, Y, Chen, Z, Peng, S et al.. Cdk1-phosphorylated Nur77 accumulates at the centrosome during mitosis to regulate the Cep192-PLK1 signaling axis. Cell Death Dis. 2026; :. doi: 10.1038/s41419-026-08870-3. PubMed PMID:42236697 .
  3. Aguado, B, Lacoste-Palasset, T, Ruffenach, G, Bauer, F, Savale, L, Humbert, M et al.. Exploring the therapeutic potential of GLP-1 receptor agonists in pulmonary arterial hypertension. ERJ Open Res. 2026;12 (3):. doi: 10.1183/23120541.01479-2025. PubMed PMID:42232566 PubMed Central PMC13224206.
  4. Wang, YZ, Li, LY, Chen, YZ, Song, DX, Liu, FY, Zhang, ZY et al.. Structural characterization and in vitro evaluation of the hypolipidemic activity of the HSP-Ia, a bioactive polysaccharide derived from hemp (Cannabis sativa L.) seeds. Carbohydr Polym. 2026;386 :125439. doi: 10.1016/j.carbpol.2026.125439. PubMed PMID:42230038 .
  5. Al-Baldawi, Z, Erzingatzian, A, Ruel, I, Brown, L, Sherman, MH, Laflamme, N et al.. Homozygous familial hypercholesterolemia (HoFH) in Canada. Atherosclerosis. 2026;418 :120792. doi: 10.1016/j.atherosclerosis.2026.120792. PubMed PMID:42229223 .
  6. Costa, E, Ross, JS, Kesselheim, AS. Orphan Designation for Drugs Approved in the United States and European Union: A Comparative Analysis. Clin Pharmacol Ther. 2026; :. doi: 10.1002/cpt.70349. PubMed PMID:42218735 .
  7. Vincze, K, Szwajda, A, Ploner, A, Karlsson, R, Kang, X, Tang, B et al.. Genetic variation in antidiabetic drug targets: associations with Parkinson's disease risk and age at onset. NPJ Parkinsons Dis. 2026;12 (1):. doi: 10.1038/s41531-026-01398-5. PubMed PMID:42209523 PubMed Central PMC13219586.
  8. Nikolcheva, T, Pagano, G, Anzures-Cabrera, J, Trundell, D, Kustermann, T, Simuni, T et al.. Efficacy and safety of intravenous prasinezumab in individuals with early-stage Parkinson's disease on stable symptomatic monotherapy (PADOVA): a phase 2b, multicentre, randomised, double-blind, placebo-controlled study. Lancet. 2026;407 (10544):2227-2240. doi: 10.1016/S0140-6736(26)00865-2. PubMed PMID:42208564 .
  9. Blandy, O, Lembo, P, Folorunso, R, Myren, KJ, Chevrou-Severac, H, Thomas, SK et al.. Use of Real-World Evidence in Health Technology Assessments of Non-Oncology Rare Disease Therapies. J Mark Access Health Policy. 2026;14 (2):. doi: 10.3390/jmahp14020032. PubMed PMID:42200984 PubMed Central PMC13214738.
  10. Fujita, M, Kamata, Y, Tanemoto, N, Takahashi, N, Tominaga, M, Takamori, K et al.. Konjac Ceramide Induces Semaphorin 3A Expression via the MAPK/AP-1 Signaling Axis and RORα in Normal Human Epidermal Keratinocytes. Biomolecules. 2026;16 (5):. doi: 10.3390/biom16050755. PubMed PMID:42194103 PubMed Central PMC13205023.
  11. Coletti, A, Calvanese, M, Cozzolino, F, Iacobucci, I, Lauro, C, Severino, A et al.. From Sea to Therapy: Development and Analytical Control of Recombinant Human CDKL5 Production in the Marine Bacterium Pseudoalteromonas haloplanktis TAC125. Mar Drugs. 2026;24 (5):. doi: 10.3390/md24050151. PubMed PMID:42188286 PubMed Central PMC13208570.
  12. Zhao, X, Zhang, M. Rare disease patients protection: theoretical discussion and institutional construction of orphan drug designation in China. Orphanet J Rare Dis. 2026; :. doi: 10.1186/s13023-026-04404-4. PubMed PMID:42185862 .
  13. Jiang, T, Zhu, Y, Liu, Z, Yi, X, Zhang, Q, Song, S et al.. ProSSF: integrating sequence, structure, and gene ontology for prediction of protein stability, interaction, and function. Mol Genet Genomics. 2026;301 (1):. doi: 10.1007/s00438-026-02433-z. PubMed PMID:42154341 .
  14. Antoni, G, Kumps, C, Berger, J, Barman-Aksözen, J, Tran, C. Access to orphan drugs in adults with inherited metabolic diseases in Switzerland: a single-center retrospective cohort study. Orphanet J Rare Dis. 2026;21 (1):. doi: 10.1186/s13023-026-04367-6. PubMed PMID:42152020 PubMed Central PMC13181876.
  15. Exavery, A, Kisyombe, D, Koler, A, Charles, J, Mallya, N, Kuhlik, E et al.. Evaluating the effects of community-based programs on viral rebound and viral suppression among HIV-positive orphaned and vulnerable children receiving antiretroviral treatment: Findings from the ACHIEVE project in Tanzania. PLoS One. 2026;21 (5):e0349141. doi: 10.1371/journal.pone.0349141. PubMed PMID:42139209 PubMed Central PMC13178892.
  16. Puopolo, GR, Rodrigues, MLA, Bulgareli, JV. Implementation of risk sharing models in access to treatment of rare diseases: an integrative review. Cien Saude Colet. 2026;31 (4):e06642024. doi: 10.1590/1413-81232026314.06642024. PubMed PMID:42138900 .
  17. Elango, R, Martin, R, Mutta, SK. Orphan drug regulation: a global comparison of the United States Food and Drug Administration, European Medicines Agency, and Japanese pharmaceuticals and medical devices agency approaches. Farm Hosp. 2026; :. doi: 10.1016/j.farma.2026.04.003. PubMed PMID:42135085 .
  18. Dreyling, M, Doorduijn, J, Giné, E, Jerkeman, M, Walewski, J, Hutchings, M et al.. Addition of autologous stem-cell transplantation to an ibrutinib-containing first-line treatment in patients aged 18-65 years with mantle cell lymphoma (TRIANGLE): 4·5-year follow-up of a three-arm, randomised, open-label, phase 3 superiority trial of the European MCL Network. Lancet. 2026;407 (10542):1953-1967. doi: 10.1016/S0140-6736(26)00362-4. PubMed PMID:42134356 .
  19. Fiorini Monteiro Novo, MA, Simões Correa Galendi, J, Cardoso Nunes Marinho, J, Nascimento Silva, S, Santos Codogno, J, Rosa Gobette, JB et al.. Systematic review protocol on evidence synthesis for health technology decisions in ultra-rare diseases. Syst Rev. 2026; :. doi: 10.1186/s13643-026-03198-7. PubMed PMID:42116179 .
  20. Zhao, Q, Liang, J, Zhou, J, Zeng, M, Zhang, W, Cao, Y et al.. Design, synthesis and structure-activity relationship study of novel indole-pyrrole scaffold compounds targeting Nur77 in colorectal tumor cells. Eur J Med Chem. 2026;314 :118927. doi: 10.1016/j.ejmech.2026.118927. PubMed PMID:42114228 .
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