PUBLICATIONS

Publications on Orphan Drugs

  1. 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. 2026;315 :119295. doi: 10.1016/j.bios.2026.119295. PubMed PMID:42832937 .
  2. 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 .
  3. 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 .
  4. 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 .
  5. 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 .
  6. 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 .
  7. 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.
  8. 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.
  9. 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 .
  10. 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 .
  11. 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.
  12. 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 .
  13. 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 .
  14. 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 .
  15. 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 .
  16. 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 .
  17. Szucs, TD. [Innovative drug development: ethical and regulatory challenges]. Ther Umsch. 2026;83 (4):151-156. doi: 10.23785/TU.2026.04.007. PubMed PMID:42678140 .
  18. Xu, M, Wang, C, An, X. Development Timelines of Innovative Drugs in China: A 15-Year Cross-Sectional Analysis of Characteristics and Influencing Factors. Clin Transl Sci. 2026;19 (9):e70702. doi: 10.1111/cts.70702. PubMed PMID:42678023 PubMed Central PMC13531702.
  19. Ilicki, J, Spicer, AJ. Rare disease benchmarks: Investment professionals' accuracy and beliefs. Drug Discov Today. 2026;31 (5):104792. doi: 10.1016/j.drudis.2026.104792. PubMed PMID:42674209 .
  20. Prusty, M, Muduli, K, Pradhan, J, Samal, AP, Tripathy, J, Reddy, KS et al.. Estrogen-related receptor α promotes breast cancer cell migration and invasion by activating transforming growth factor β signaling. Anticancer Drugs. 2026; :. doi: 10.1097/CAD.0000000000001840. PubMed PMID:42669034 .
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