PUBLICATIONS

Publications on Orphan Drugs

  1. 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 .
  2. 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.
  3. 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.
  4. 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 .
  5. 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 .
  6. 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 .
  7. 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 .
  8. 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 .
  9. Yoshida, H, Suto, T, Hirano, H, Osada, H, Nunomura, K, Lin, B et al.. Novel Ligands for the Orphan Receptor GPR151 Modulate Morphine Action. Genes Cells. 2026;31 (3):e70119. doi: 10.1111/gtc.70119. PubMed PMID:42104714 PubMed Central PMC13156533.
  10. Summar, ML, Woodcock, J. Supply and Demand in the Mathematics of Rare Disease Drug Development: Why Choosing the Right Model Is Crucial. Clin Transl Sci. 2026;19 (5):e70551. doi: 10.1111/cts.70551. PubMed PMID:42104173 PubMed Central PMC13156073.
  11. Schihada, H, Shahraki, A, Turku-Metsänen, A, Rath, M, Wirth, L, Nemec, K et al.. GPR3 Ligands Discovered through Combined Virtual and Conformational Biosensor-Based Screening. J Am Chem Soc. 2026;148 (19):20285-20295. doi: 10.1021/jacs.6c06780. PubMed PMID:42096618 .
  12. Penninx, BMF, Tas, SW, Timmers, L, van de Kar, NCAJ, Huisman, A, van den Berg, D et al.. Rational use of expensive medicines in the Netherlands: strategies to improve effectiveness and reduce burden on patients and society - a narrative review. BMC Med. 2026; :. doi: 10.1186/s12916-026-04897-7. PubMed PMID:42062879 .
  13. Brahmi, M, Romeo, C, Meurgey, A, Dupont, M, Dufresne, A. Desmoplastic small round cell tumor. Curr Opin Oncol. 2026; :. doi: 10.1097/CCO.0000000000001239. PubMed PMID:42054145 .
  14. Caimmi, M, Canali, B, Ciarlo, A, Lombardi, G, Vassallo, C, Candelora, L et al.. [Not Available]. Glob Reg Health Technol Assess. 2026;13 :87-94. doi: 10.33393/grhta.2026.3717. PubMed PMID:42038443 PubMed Central PMC13103655.
  15. Pintado, S, Fernandes, A, Bührman, EAM, IntHout, J, Oliveira-Martins, S. Regulatory innovation for drug repurposing: Ten proposals to accelerate access and safety. Drug Discov Today. 2026;31 (3):104682. doi: 10.1016/j.drudis.2026.104682. PubMed PMID:42035866 .
  16. Balg, C, Frangen, K, Brockhaus, AC, Sturtz, S, Schwalm, A, Mostardt, S et al.. Price development of orphan drugs in Germany after market access and subsequent assessments: a descriptive analysis. Eur J Health Econ. 2026; :. doi: 10.1007/s10198-026-01926-3. PubMed PMID:42018120 .
  17. Tiwari, HD, Sahoo, MK, Murali, MR, Tiwari, R, Manjusha, K, Prashant, MC et al.. Orphan Drugs and Diseases: A Systematic Review. J Pharm Bioallied Sci. 2026;18 (2):93-95. doi: 10.4103/jpbs.jpbs_937_25. PubMed PMID:42005478 PubMed Central PMC13086369.
  18. Okuyama, R. National differences in drug discovery and the role of startups: Insights from cross-national analysis of Phase III candidates. Drug Discov Today. 2026;31 (3):104672. doi: 10.1016/j.drudis.2026.104672. PubMed PMID:42000127 .
  19. 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;11 (6):463-494. doi: 10.1016/S2468-1253(26)00011-7. PubMed PMID:41990758 .
  20. 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.
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