PUBLICATIONS

Publications on Orphan Drugs

  1. Gagnon, MA. Commentary: Which Principles Should Apply for a National Strategy on Rare Diseases?. Healthc Policy. 2024;19 (4):27-31. doi: 10.12927/hcpol.2024.27353. PubMed PMID:39229660 .
  2. Lexchin, J, Sirrs, S. Federal Funding for Expensive Drugs for Rare Diseases: How Do We Pick and Choose?. Healthc Policy. 2024;19 (4):19-26. doi: 10.12927/hcpol.2024.27334. PubMed PMID:39229659 .
  3. Guignabert, C, Aman, J, Bonnet, S, Dorfmüller, P, Olschewski, AJ, Pullamsetti, S et al.. Pathology and pathobiology of pulmonary hypertension: current insights and future directions. Eur Respir J. 2024; :. doi: 10.1183/13993003.01095-2024. PubMed PMID:39209474 .
  4. Varshney, S, Kumar, D, Choudhary, R, Gupta, A, Beg, M, Shankar, K et al.. Flavopiridol inhibits adipogenesis and improves metabolic homeostasis by ameliorating adipose tissue inflammation in a diet-induced obesity model. Biomed Pharmacother. 2024;179 :117330. doi: 10.1016/j.biopha.2024.117330. PubMed PMID:39208666 .
  5. Nuijten, M, Capri, S. Valuation of Medical Innovation Handling with Uncertainty and Risk. J Mark Access Health Policy. 2024;12 (3):199-208. doi: 10.3390/jmahp12030016. PubMed PMID:39193543 PubMed Central PMC11348113.
  6. de Alencar Morais Lima, W, de Souza, JG, García-Villén, F, Loureiro, JL, Raffin, FN, Fernandes, MAC et al.. Next-generation pediatric care: nanotechnology-based and AI-driven solutions for cardiovascular, respiratory, and gastrointestinal disorders. World J Pediatr. 2024; :. doi: 10.1007/s12519-024-00834-x. PubMed PMID:39192003 .
  7. Costa, E, Girotti, S, Mathieu, C, Castellani, C, Ross, JS, Taylor-Cousar, JL et al.. Differential times of submission and approval of CFTR modulators for the treatment of Cystic Fibrosis in the United States and the European Union. J Cyst Fibros. 2024; :. doi: 10.1016/j.jcf.2024.08.002. PubMed PMID:39183127 .
  8. Koban, MU, Hartmann, M, Amexis, G, Franco, P, Huggins, L, Shah, I et al.. Targeted therapies, novel antibodies, and immunotherapies in advanced non-small cell lung cancer: clinical evidence and drug approval patterns. Clin Cancer Res. 2024; :. doi: 10.1158/1078-0432.CCR-24-0741. PubMed PMID:39177967 .
  9. Obst, CS, Seifert, R. Updated analysis of the prescription and evaluation of protein kinase inhibitors for oncology in Germany. Naunyn Schmiedebergs Arch Pharmacol. 2024; :. doi: 10.1007/s00210-024-03377-0. PubMed PMID:39177786 .
  10. Xia, Y, Chen, H, Qin, J, Zhang, W, Gao, H, Long, X et al.. The phthalide compound tokinolide B from Angelica sinensis exerts anti-inflammatory effects through Nur77 binding. Phytomedicine. 2024;133 :155925. doi: 10.1016/j.phymed.2024.155925. PubMed PMID:39173278 .
  11. Schoenmakers, DH, van den Berg, S, Timmers, L, Adang, LA, Bäumer, T, Bosch, A et al.. Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases. Neurology. 2024;103 (6):e209743. doi: 10.1212/WNL.0000000000209743. PubMed PMID:39173102 PubMed Central PMC11379353.
  12. Siziya, IN, Lim, HJ, Baek, S, Lee, S, Seo, MJ. Mannosidase-inhibiting iminosugar production by recombinant Corynebacterium glutamicum harboring the 1-deoxynojirimycin biosynthetic gene cluster. Int J Biol Macromol. 2024;278 (Pt 2):134858. doi: 10.1016/j.ijbiomac.2024.134858. PubMed PMID:39163968 .
  13. Abdallah, K, Huys, I, Claes, KJ, Simoens, S. Budget Impact of Disease-Modifying Treatments and a CRISPR Gene-Edited Therapy for Sickle Cell Disease. Clin Drug Investig. 2024;44 (8):611-627. doi: 10.1007/s40261-024-01384-w. PubMed PMID:39134876 .
  14. Xiang, Z, Zhang, B, Cao, S, Cao, L, Li, L, Huang, D et al.. SPH7854, a gut-limited RORγt antagonist, ameliorates TNBS-induced experimental colitis in rat. Int Immunopharmacol. 2024;140 :112884. doi: 10.1016/j.intimp.2024.112884. PubMed PMID:39133959 .
  15. Klein, AL, Wang, TKM, Cremer, PC, Abbate, A, Adler, Y, Asher, C et al.. Pericardial Diseases: International Position Statement on New Concepts and Advances in Multimodality Cardiac Imaging. JACC Cardiovasc Imaging. 2024;17 (8):937-988. doi: 10.1016/j.jcmg.2024.04.010. PubMed PMID:39111992 .
  16. Panmanee, J, Charoensutthivarakul, S, Cheng, CW, Promthep, K, Mukda, S, Prasertporn, T et al.. A Complex Interplay Between Melatonin and RORβ: RORβ is Unlikely a Putative Receptor for Melatonin as Revealed by Biophysical Assays. Mol Neurobiol. 2024; :. doi: 10.1007/s12035-024-04395-y. PubMed PMID:39105871 .
  17. Clifford, KA, Levine, AA, Enright, DE, Neumann, PJ, Chambers, JD. The Health Benefits, Costs, and Cost-Effectiveness of Ultraorphan Drugs. Value Health. 2024; :. doi: 10.1016/j.jval.2024.07.005. PubMed PMID:39094687 .
  18. Alipour-Haris, G, Liu, X, Acha, V, Winterstein, AG, Burcu, M. Real-world evidence to support regulatory submissions: A landscape review and assessment of use cases. Clin Transl Sci. 2024;17 (8):e13903. doi: 10.1111/cts.13903. PubMed PMID:39092896 PubMed Central PMC11295294.
  19. Mishra, S, Venkatesh, MP. Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges. Orphanet J Rare Dis. 2024;19 (1):285. doi: 10.1186/s13023-024-03146-5. PubMed PMID:39085891 PubMed Central PMC11292868.
  20. Wang, Y, Zhou, N, Li, B, Lv, Z, Duan, S, Li, X et al.. Utilization and affordability of health insurance coverage for rare disease drugs in a first-tier city in Northeast China from 2018 to 2021: a study based on the health insurance claims database. Int J Equity Health. 2024;23 (1):151. doi: 10.1186/s12939-024-02225-0. PubMed PMID:39085851 PubMed Central PMC11290155.
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