PUBLICATIONS

Publications on Orphan Drugs

  1. Jain, U, Polkampally, S, Jadav, AA, Jain, B, Jain, P, Haumschild, R et al.. Value-based care contracting for pharmacy drugs: opportunity for the United States. Value Health. 2026; :. doi: 10.1016/j.jval.2026.08.014. PubMed PMID:42918324 .
  2. Yamaguchi, S, Nakatani, E, Asada, R. Investigator-Initiated Trials as a Clinical Development Approach for Orphan Drugs Compared with Industry-Sponsored Trials in Japan. Ther Innov Regul Sci. 2026; :. doi: 10.1007/s43441-026-01063-2. PubMed PMID:42909798 .
  3. Bowen, HP, O'Loughlin, G, Schleicher, C, Schulthess, DG. The Inflation Reduction Act's Modeled Impacts upon Late-Stage R&D. Ther Innov Regul Sci. 2026; :. doi: 10.1007/s43441-026-01061-4. PubMed PMID:42902123 .
  4. Arora, R, Gambhir, S, Bhardwaj, A, Gupta, P. NR4A2: a next-generation drug target orchestrating dynamic gene regulation and protein interactions. Drug Discov Today. 2026; :104837. doi: 10.1016/j.drudis.2026.104837. PubMed PMID:42901401 .
  5. Avouac, J, Jouret, M, Bitoun, S, Borie, R, Cornec, D, Devauchelle-Pensec, V et al.. Innovative therapies in rheumatoid arthritis: CAR T cells and bispecific antibodies. Lancet Rheumatol. 2026;8 (11):e908-e920. doi: 10.1016/S2665-9913(26)00184-0. PubMed PMID:42896926 .
  6. Blonda, A, Ollendorf, DA, Huys, I, Denier, Y, Simoens, S. Making trade-offs explicit: development and pilot of a Belgian MCDA framework for orphan drug appraisal. Int J Technol Assess Health Care. 2026;42 (1):e96. doi: 10.1017/S0266462326104127. PubMed PMID:42894980 .
  7. Costa, E, Kesselheim, AS. The Impact of Semantics in Medicines Regulation. J Law Med Ethics. 2026; :1-3. doi: 10.1017/jme.2026.10293. PubMed PMID:42894459 .
  8. Cabar, CK, Cabar, FR. Therapeutic orphans no more: Revisiting the ethical justification for excluding pregnant women from pharmaceutical clinical trials. Int J Gynaecol Obstet. 2026; :. doi: 10.1002/ijgo.71402. PubMed PMID:42885831 .
  9. Kim, JY, Shin, EY, Park, JW, Lee, SS, Lee, YJ, Jung, YJ et al.. Frequency, causes, and duration of drug shortages in South Korea, 2018-2024: a nationwide descriptive analysis of regulatory reporting data and temporal patterns across the COVID-19 pandemic. Front Pharmacol. 2026;17 :1931826. doi: 10.3389/fphar.2026.1931826. PubMed PMID:42865490 PubMed Central PMC13655078.
  10. Siddiqui, L, Khan, KM, Saleem, RSZ. An Extensive Review of Recent Progress In Kinase-targeting Antimalarial Drug Discovery. Mini Rev Med Chem. 2026; :. doi: 10.2174/0113895575502571260924050214. PubMed PMID:42864805 .
  11. Rahe-Meyer, N, Rodrigues, R, Fries, D, Steffen Schmidt, D, Faraoni, D. The role of fibrinogen concentrate in the reduction of allogeneic blood product requirements: a systematic review and meta-analysis. Br J Anaesth. 2026;137 (5):1386-1403. doi: 10.1016/j.bja.2026.07.047. PubMed PMID:42864190 .
  12. Tabarzad, M, Heidari, M, Norouzi, B. Bioinformatics-driven discovery of bioactive natural peptides from cyanobacterial genomes. Bioresour Technol. 2026;464 (Pt C):136049. doi: 10.1016/j.biortech.2026.136049. PubMed PMID:42863716 .
  13. Fraser, AG, Byrne, RA, Dequeker, EMC, Kjærsgaard-Andersen, P, Kolaček, S, Melvin, T et al.. Empowering regulatory science for medical devices in Europe. Lancet Reg Health Eur. 2026;71 :101876. doi: 10.1016/j.lanepe.2026.101876. PubMed PMID:42859472 PubMed Central PMC13652854.
  14. Kim, J, Ha, T, Park, S, Kang, D, Lee, S, Lee, S et al.. Nanopore sensing with machine learning-assisted analysis for real-time single-molecule detection of MDM2-p53TAD interactions. Biosens Bioelectron. 2027;315 :119295. doi: 10.1016/j.bios.2026.119295. PubMed PMID:42832937 .
  15. Li, X, Yu, X, Huang, K, Luo, S, Li, J, Song, C et al.. NR2C2 suppresses breast cancer progression and promotes T-Cell infiltration in the tumor microenvironment. J Mol Histol. 2026;57 (5):. doi: 10.1007/s10735-026-10984-z. PubMed PMID:42828550 .
  16. Adolf, J, Raykov, G. Orphaned by an Evidentiary Standard: How Efficacy Review Emptied the Anabolic-Androgenic Steroid Market, and What Doping Control Inherited. Drug Test Anal. 2026; :. doi: 10.1002/dta.70159. PubMed PMID:42823853 .
  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.2026.08.009. PubMed PMID:42810926 .
  18. Papa, R, Rhee, S, Delai, P, Zhang, K, Balcázar, IB, Rethanavelu, K et al.. Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2026;408 (10564):1583-1592. doi: 10.1016/S0140-6736(26)01599-0. PubMed PMID:42805227 .
  19. Wang, X, Du, R, Wang, Y, Siddique, AZMF, Li, B, Wang, Q et al.. Nesfatin-1 as a Cardiovascular-Metabolic-Immune Integrator: Insights from Cellular Cross-talk to Precision Medicine. J Cardiovasc Transl Res. 2026;19 (1):. doi: 10.1007/s12265-026-10830-3. PubMed PMID:42802210 .
  20. Torre Flores, LP, Jalink, PJ, Fuentes-Mateos, R, Erasmus, M, van Oorschot, ME, Gan, T et al.. Defective alveolar regeneration in emphysema: identifying dysfunctional cellular crosstalk and novel therapeutic opportunities for tissue repair. Eur Respir Rev. 2026;35 (181):. doi: 10.1183/16000617.0090-2026. PubMed PMID:42778202 PubMed Central PMC13598616.
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