PUBLICATIONS

Publications on Orphan Drugs

  1. 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.
  2. 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 .
  3. Tabarzad, M, Heidari, M, Norouzi, B. Bioinformatics-driven discovery of bioactive natural peptides from cyanobacterial genomes. Bioresour Technol. 2026; :136049. doi: 10.1016/j.biortech.2026.136049. PubMed PMID:42863716 .
  4. 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.
  5. 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 .
  6. 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 .
  7. 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 .
  8. 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 .
  9. 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; :. doi: 10.1016/S0140-6736(26)01599-0. PubMed PMID:42805227 .
  10. 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 .
  11. 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.
  12. Li, KH, Cousin, EM, Sullivan, SD. Which drugs will Medicare negotiate next? Predicting selected drugs for Initial Price Applicability Year 2029. J Manag Care Spec Pharm. 2026;32 (10):1189-1200. doi: 10.18553/jmcp.2026.26277. PubMed PMID:42776848 PubMed Central PMC13621897.
  13. Swenson, W, Schroeder, Z. Rural Cancer Care Delivery in the United States: A 2026 Update on Disparities, Vulnerable Populations, and Emerging Models. JCO Oncol Pract. 2026; :OP2600440. doi: 10.1200/OP-26-00440. PubMed PMID:42766808 .
  14. Distler, JHW, Assassi, S, Gabrielli, A, Allanore, Y, Chung, L, Cutolo, M et al.. Conceptual framework for the definition of disease modification in systemic sclerosis and potential implications for drug development and clinical study design. Ann Rheum Dis. 2026; :. doi: 10.1016/j.ard.2026.08.006. PubMed PMID:42736088 .
  15. Mokrysheva, NG, Bardymova, TP, Bibik, EE, Eremkina, AK, Karonova, TL, Kovaleva, EV et al.. [Resolution of the Joint Meeting of the Expert Council "Hypoparathyroidism in the Era of Complex Problems and Breakthrough Technologies"]. Probl Endokrinol (Mosk). 2026;72 (4):10-17. doi: 10.14341/probl13805. PubMed PMID:42733369 PubMed Central PMC13585288.
  16. Vargas, C, Goodall, S, Espinoza, M, De Abreu Lourenco, R. Rare disease definitions across regulatory and reimbursement systems: policy objectives and implications for patient access. Health Policy. 2026;174 :105761. doi: 10.1016/j.healthpol.2026.105761. PubMed PMID:42727519 .
  17. Río Gómez, PD, Caíña López, S, Dávila Pousa, MDC. Regulation and analysis of the access to orphan drugs in the pediatric population in Spain. An Pediatr (Engl Ed). 2026;105 (3):504315. doi: 10.1016/j.anpede.2026.504315. PubMed PMID:42716904 .
  18. Hitsch, F, Seifert, R. Analysis of marketing authorizations, withdrawals, and refusals by the European Medicines Agency (EMA): trends in approval procedures 1995-2025. Naunyn Schmiedebergs Arch Pharmacol. 2026; :. doi: 10.1007/s00210-026-05887-5. PubMed PMID:42706333 .
  19. Rodriguez, A, Krishnan, V, Polli, JE, Schwendeman, A. Importance of Single Global Regulatory Standards for the Assessment of Generic and Biosimilar Medicines to Advance Patient Access. AAPS J. 2026;28 (5):. doi: 10.1208/s12248-026-01272-5. PubMed PMID:42693311 .
  20. Alshaikh, RA, Cronin, DJ, Hughes, DA, O'Driscoll, CM. Navigating patient access pathways for non-viral RNA-based therapeutics: a comparative review of health technology assessment and reimbursement pathways across six countries. Eur J Pharm Sci. 2026;227 :107652. doi: 10.1016/j.ejps.2026.107652. PubMed PMID:42685807 .
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