PUBLICATIONS

Publications on Orphan Drugs

  1. Zhai, C, Wang, J, Xia, B, Guo, Y, Zhang, Y, Jiao, Y et al.. Awareness of Duchenne muscular dystrophy among medical staff in China: a multicenter cross-sectional survey. Front Pediatr. 2026;14 :1735961. doi: 10.3389/fped.2026.1735961. PubMed PMID:42290726 PubMed Central PMC13253694.
  2. Li, X, Qiao, S, Li, Z, Zhou, S, Wang, J, Wang, B et al.. Predicting protein-protein interaction sites based on dynamic perception mechanism within a hierarchical E(n)-equivariant graph. Brief Bioinform. 2026;27 (3):. doi: 10.1093/bib/bbag304. PubMed PMID:42289050 .
  3. Malherbe, HL, Odendaal, S, Kukava, A, Lucano, C. The estimated burden of rare diseases in South Africa using Orphanet: an epidemiological analysis. Orphanet J Rare Dis. 2026; :. doi: 10.1186/s13023-026-04444-w. PubMed PMID:42277930 .
  4. Shi, Q, Wang, G, Zhang, T, Zhao, Y, Meng, F, Feng, K et al.. Melatonin-Mediated Modulation of Polyamines Enhances Drought Tolerance in Foxtail Millet (Setaria italica L.). J Pineal Res. 2026;78 (4):e70157. doi: 10.1111/jpi.70157. PubMed PMID:42267978 .
  5. 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 .
  6. 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 .
  7. 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.
  8. 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 .
  9. 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 .
  10. 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 .
  11. 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.
  12. 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 .
  13. 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.
  14. 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.
  15. Yang, T, An, Q, Lin, S, Sun, J, Wang, Y, Chen, H et al.. Design and Evaluation of 68Ga-Labeled Peptide-Based PET Probes for Noninvasive Imaging of ROR1 Expression in Tumor Models. J Med Chem. 2026;69 (11):13941-13952. doi: 10.1021/acs.jmedchem.6c01128. PubMed PMID:42191668 .
  16. 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.
  17. 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 .
  18. 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 .
  19. 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.
  20. Hosseini, F, Mobinizadeh, M, Padula, WV. Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases. Appl Health Econ Health Policy. 2026;24 (4):647-654. doi: 10.1007/s40258-026-01048-0. PubMed PMID:42143656 PubMed Central PMC13260230.
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