PUBLICATIONS

Publications on Orphan Drugs

  1. Li, Y, Zhang, B, Zhang, Z, Yan, W, Wang, H, Xu, X et al.. Inhibition of the RORC/GPX4 mediated ferroptosis regulatory axis suppresses tumor growth and alleviates enzalutamide resistance in prostate cancer. Cell Mol Biol Lett. 2026;31 (1):11. doi: 10.1186/s11658-025-00846-z. PubMed PMID:41580593 PubMed Central PMC12837284.
  2. Alperin, C, Nguyen, N, Duffey, M, Clark, EH. Priced Out of Treatment: The Exorbitant Cost of Antiparasitic Drugs in the United States. Open Forum Infect Dis. 2026;13 (1):ofaf784. doi: 10.1093/ofid/ofaf784. PubMed PMID:41567261 PubMed Central PMC12817971.
  3. Camilleri, M, Parkman, HP. Review Article: Drug Approval for Gastroparesis-Suggestions for Improving the Process for Positive Results. Aliment Pharmacol Ther. 2026; :. doi: 10.1111/apt.70544. PubMed PMID:41566871 .
  4. Martin, P, Ding, J, Hanchard, M. Why is the pharmaceutical industry investing in targeted therapies? The emergence of "premium pharma". Soc Sci Med. 2025;392 :118888. doi: 10.1016/j.socscimed.2025.118888. PubMed PMID:41548522 .
  5. Hong, EH, Hyeong, J, Ahn, JH, Han, Y, Kim, S, Kim, SM et al.. Limosilactobacillus reuteri alleviates psoriasis via aryl hydrocarbon receptor-mediated regulation of Interkeukin-17A. Int Immunopharmacol. 2026;172 :116194. doi: 10.1016/j.intimp.2026.116194. PubMed PMID:41548440 .
  6. Minder, AE, Barman-Aksözen, J. From darkness to light: Case report on afamelanotide-treatment in a 9-year-old child with erythropoietic protoporphyria. JAAD Case Rep. 2026;68 :6-8. doi: 10.1016/j.jdcr.2025.11.022. PubMed PMID:41542313 PubMed Central PMC12800419.
  7. Havlik, J, Isaac, S, Radovan, C, Ostacher, MJ, Smith, D, Rhee, TG et al.. Innovation in Psychiatric Drug Development: A Quantitative Analysis of FDA-Approved Psychiatric Drugs, 2012-2024. J Clin Psychiatry. 2026;87 (1):. doi: 10.4088/JCP.25m16063. PubMed PMID:41532845 .
  8. Avxentyev, NA, Makarov, AS, Germanenko, OY, Vlodavets, DV, Kuzenkova, LM. [Pharmacoeconomic evaluation of spinal muscular atrophy therapy in patients with four SMN2 copies diagnosed through newborn screening]. Zh Nevrol Psikhiatr Im S S Korsakova. 2025;125 (12):188-199. doi: 10.17116/jnevro2025125121188. PubMed PMID:41524367 .
  9. Khalifa, A, Malden, DE, Ratmann, O, Chen, Y, Grabowski, MK, Chang, LW et al.. Incidence and prevalence of orphanhood in Rakai, Uganda: a population-based cohort study, 1995-2022. Lancet Glob Health. 2026;14 (2):e251-e260. doi: 10.1016/S2214-109X(25)00440-1. PubMed PMID:41519154 .
  10. Auernhammer, CJ, Wang, K, Maccio, U, Knösel, T, Hungbauer, MP, Schilbach, K et al.. Personalized drug screening and risk assessment in patient-derived gastroenteropancreatic neuroendocrine neoplasms. J Clin Endocrinol Metab. 2026; :. doi: 10.1210/clinem/dgaf705. PubMed PMID:41518601 .
  11. Lippi, D, Varotto, E, Galassi, FM, Baldanzi, F. Understanding and teaching rare diseases: from historical origins to modern classification. Postgrad Med J. 2026; :. doi: 10.1093/postmj/qgaf191. PubMed PMID:41518138 .
  12. Reid, B, Cohen, JT, Lin, PJ, Neumann, PJ. Communication of launch prices by drug companies, 2022-2024. Am J Manag Care. 2025;31 (12):e351-e352. doi: 10.37765/ajmc.2025.89838. PubMed PMID:41512263 .
  13. Walraven, J, Kaveh, M, Uyl-de Groot, C. Methodological challenges in Dutch HTA of non-oncological orphan drugs: a retrospective analysis and price comparison using different pricing models. Orphanet J Rare Dis. 2026; :. doi: 10.1186/s13023-025-04181-6. PubMed PMID:41501932 .
  14. Höger, P, Ries, M, Olivares Rivera, A, Ersöz, H, Buschulte, K, Fähndrich, S et al.. Advances in orphan drug development for alpha-1 antitrypsin deficiency: a 2025 update from the FDA and EMA. Ther Adv Respir Dis. 2026;20 :17534666251411227. doi: 10.1177/17534666251411227. PubMed PMID:41499187 PubMed Central PMC12779913.
  15. Perlstein, I, Cherniakov, I, Elgart, A, Gomeni, R, Gutman, D, Merenlender Wagner, A et al.. Population Pharmacokinetic Model-Based Dose Selection of Extended-Release Injectable Olanzapine (TV-44749) for Subcutaneous Use in Phase 3 Clinical Trial in Adults with Schizophrenia. J Clin Pharmacol. 2026;66 (1):e70144. doi: 10.1002/jcph.70144. PubMed PMID:41496255 PubMed Central PMC12775547.
  16. Borowsky, B, Ramos, H, Caputo, A, Hartmann, A, Faller, T, Peters, T et al.. Oral splicing modulator branaplam in Huntington's disease: a phase 2 randomized controlled trial. Nat Med. 2026;32 (1):103-112. doi: 10.1038/s41591-025-04117-4. PubMed PMID:41491108 PubMed Central PMC12823413.
  17. Goto, R, Sengoku, S, Kodama, K. [Programmed Medical Device (SaMD) and Orphan Drug Innovation Processes and Industrial Systems]. Yakugaku Zasshi. 2026;146 (1):35-46. doi: 10.1248/yakushi.25-00140-3. PubMed PMID:41485960 .
  18. Kodama, K. [Current Status of Drug Lag in Rare Disease Treatment and a Multifaceted Approach to Resolving It]. Yakugaku Zasshi. 2026;146 (1):25-34. doi: 10.1248/yakushi.25-00140-2. PubMed PMID:41485959 .
  19. Enya, K. [Increasing Orphan Drug Loss in Japan: Trends and R&D Strategy for Rare Diseases]. Yakugaku Zasshi. 2026;146 (1):15-24. doi: 10.1248/yakushi.25-00140-1. PubMed PMID:41485958 .
  20. Santamaria, X, Pardo-Figuerez, M, González-Fernández, J, Querol, S, Rodríguez, L, Valcárcel, D et al.. Autologous cell therapy with CD133+ bone marrow-derived stem cells for Asherman Syndrome: a phase 1/2 trial. Nat Commun. 2026; :. doi: 10.1038/s41467-025-67850-x. PubMed PMID:41484096 .
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