PUBLICATIONS

Publications on Orphan Drugs

  1. Gangwal, A, Lavecchia, A. AI-Driven Drug Discovery for Rare Diseases. J Chem Inf Model. 2024; :. doi: 10.1021/acs.jcim.4c01966. PubMed PMID:39689164 .
  2. Gensapa, R, Pandey, V, Kumar Saha, S, Kumar Mukherjee, S, Jha, A. Inter-Relational Dynamics of Factors Affecting the Emergence of Orphan Drugs. Ann Pharm Fr. 2024; :. doi: 10.1016/j.pharma.2024.12.005. PubMed PMID:39675421 .
  3. Tomsen-Melero, J, Moltó-Abad, M, Merlo-Mas, J, Díaz-Riascos, ZV, Cristóbal-Lecina, E, Soldevila, A et al.. Targeted nanoliposomes to improve enzyme replacement therapy of Fabry disease. Sci Adv. 2024;10 (50):eadq4738. doi: 10.1126/sciadv.adq4738. PubMed PMID:39671483 .
  4. Global Nutrition Target Collaborators. Global, regional, and national progress towards the 2030 global nutrition targets and forecasts to 2050: a systematic analysis for the Global Burden of Disease Study 2021. Lancet. 2024; :. doi: 10.1016/S0140-6736(24)01821-X. PubMed PMID:39667386 .
  5. Pizzamiglio, C, Stefanetti, RJ, McFarland, R, Thomas, N, Ransley, G, Hugerth, M et al.. Optimizing rare disorder trials: a phase 1a/1b randomized study of KL1333 in adults with mitochondrial disease. Brain. 2024; :. doi: 10.1093/brain/awae308. PubMed PMID:39657714 .
  6. Kakkilaya, A, Shahzad, M, Bourgeois, FT. FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023. JAMA Pediatr. 2024; :. doi: 10.1001/jamapediatrics.2024.5280. PubMed PMID:39652344 .
  7. Mennini, FS, Cicchetti, A, Sciattella, P, Rumi, F, Zanuzzi, M, Carletto, A et al.. Determinants of the Financial Impact of Orphan Drugs in Italy: Differences Between Expected and Observed Pharmaceutical Expenditure. Drugs Real World Outcomes. 2024; :. doi: 10.1007/s40801-024-00463-w. PubMed PMID:39644444 .
  8. Ma, X, Chen, LN, Liao, M, Zhang, L, Xi, K, Guo, J et al.. Molecular insights into the activation mechanism of GPR156 in maintaining auditory function. Nat Commun. 2024;15 (1):10601. doi: 10.1038/s41467-024-54681-5. PubMed PMID:39638804 PubMed Central PMC11621567.
  9. Merz, M, Dima, D, Hashmi, H, Ahmed, N, Stölzel, F, Holderried, TAW et al.. Bispecific antibodies targeting BCMA or GPRC5D are highly effective in relapsed myeloma after CAR T-cell therapy. Blood Cancer J. 2024;14 (1):214. doi: 10.1038/s41408-024-01197-2. PubMed PMID:39632797 PubMed Central PMC11618392.
  10. Jakubowski, S, Holko, P, Nowak, R, Warmuth, M, Dooms, M, Salminen, O et al.. Clinical and non-clinical aspects of reimbursement policy for orphan drugs in selected European countries. Front Pharmacol. 2024;15 :1498386. doi: 10.3389/fphar.2024.1498386. PubMed PMID:39629081 PubMed Central PMC11611580.
  11. Kranz, P, McGauran, N, Ünal, C, Kaiser, T. Results of health technology assessments of orphan drugs in Germany-lack of added benefit, evidence gaps, and persisting unmet medical needs. Int J Technol Assess Health Care. 2024;40 (1):e68. doi: 10.1017/S026646232400062X. PubMed PMID:39623907 .
  12. Goi, N, Iwata, F, Sugihara, Y, Higa, S, Chikama, T. An Open-Label, Phase III Study to Assess the Efficacy and Safety of Cysteamine Ophthalmic Solution 0.55% in Japanese Cystinosis Patients. Clin Ophthalmol. 2024;18 :3457-3471. doi: 10.2147/OPTH.S479770. PubMed PMID:39618989 PubMed Central PMC11606150.
  13. Mishra, S, Bhat, D, Venkatesh, MP. Navigating health policies and programs in India: exploring opportunities to improve rare disease management and orphan drug research. Orphanet J Rare Dis. 2024;19 (1):446. doi: 10.1186/s13023-024-03377-6. PubMed PMID:39614301 PubMed Central PMC11606027.
  14. Crass, RL, Smith, B, Adriaens, S, Chapel, S, Langdon, G. Population Pharmacokinetic and Pharmacokinetic/Pharmacodynamic Analyses of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria. Drugs R D. 2024;24 (4):563-573. doi: 10.1007/s40268-024-00500-7. PubMed PMID:39612158 PubMed Central PMC11652457.
  15. Karres, D, Pino-Barrio, MJ, Benchetrit, S, Benda, N, Cochat, P, Galluzzo, S et al.. Evidence generation throughout paediatric medicines life cycle: findings from collaborative work between European Medicines Agency (EMA) and EUnetHTA on use of extrapolation. Br J Pharmacol. 2024; :. doi: 10.1111/bph.17396. PubMed PMID:39574299 .
  16. Yang, W, Jin, M, Wang, Y, Zhao, H, Wang, X, Guo, Y et al.. NR1D1 activation alleviates inflammatory response through inhibition of IL-6 expression in bovine endometrial epithelial cells. Int J Biol Macromol. 2024;283 (Pt 4):137642. doi: 10.1016/j.ijbiomac.2024.137642. PubMed PMID:39551321 .
  17. Sun, SL, Xu, HJ, Jiang, XL, Zhou, J, Shi, W, Wang, XJ et al.. Discovery of 1-(Phenylsulfonyl)-1,2,3,4-tetrahydroquinoline Derivative as Orally Bioavailable and Safe RORγt Inverse Agonists for Potential Treatment of Rheumatoid Arthritis. J Med Chem. 2024;67 (22):20315-20342. doi: 10.1021/acs.jmedchem.4c01727. PubMed PMID:39546350 .
  18. Igarashi, A, Tanaka, S, De Moor, R, Li, N, Hirozane, M, Wu, DB et al.. Indirect Treatment Comparisons in Healthcare Decision Making: A Targeted Review of Regulatory Approval, Reimbursement, and Pricing Recommendations Globally for Oncology Drugs in 2021-2023. Adv Ther. 2024; :. doi: 10.1007/s12325-024-03013-6. PubMed PMID:39531127 .
  19. Rose, K, Grant-Kels, JM, Striano, P. Therapeutic orphans, off-label, pediatric drug development: towards reasonable pharmacotherapy for minors. Expert Opin Pharmacother. 2024;25 (18):2375-2384. doi: 10.1080/14656566.2024.2426678. PubMed PMID:39526437 .
  20. Sampei, Z, Haraya, K, Gan, SW, Muraoka, M, Hayasaka, A, Fukuzawa, T et al.. Beyond Recycling Antibodies: Crovalimab's Molecular Design Enables Four-Weekly Subcutaneous Injections for PNH Treatment. Int J Mol Sci. 2024;25 (21):. doi: 10.3390/ijms252111679. PubMed PMID:39519232 PubMed Central PMC11546984.
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