PUBLICATIONS

Publications on Orphan Drugs

  1. 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 .
  2. 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.
  3. 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.
  4. 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; :. doi: 10.1007/s40268-024-00500-7. PubMed PMID:39612158 .
  5. 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 .
  6. 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 .
  7. 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 .
  8. Rose, K, Grant-Kels, JM, Striano, P. Therapeutic orphans, off-label, pediatric drug development: towards reasonable pharmacotherapy for minors. Expert Opin Pharmacother. 2024; :1-10. doi: 10.1080/14656566.2024.2426678. PubMed PMID:39526437 .
  9. 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.
  10. Domike, R, Raju, GK, Sullivan, J, Kennedy, A. Expediting treatments in the 21st century: orphan drugs and accelerated approvals. Orphanet J Rare Dis. 2024;19 (1):418. doi: 10.1186/s13023-024-03398-1. PubMed PMID:39516878 PubMed Central PMC11549740.
  11. Silva, MA, Williams, S, Hauert, S, Ovadia, B, Gupta, I, Waldmeier, L et al.. Tumour assessment of ROR1 levels in various adult leukaemia and lymphoma types. PLoS One. 2024;19 (11):e0313026. doi: 10.1371/journal.pone.0313026. PubMed PMID:39495778 PubMed Central PMC11534244.
  12. Navarro, P, Beato, C, Rodriguez-Moreno, JF, Ruiz-Llorente, S, Mielgo, X, Pineda, E et al.. Prospective study of the real impact of fusion centered genomic assays in patient management in a national collaborative group: the GETHI-XX-16 study. Clin Transl Oncol. 2024; :. doi: 10.1007/s12094-024-03745-5. PubMed PMID:39485597 .
  13. Deng, D, Wu, Y, Wu, K, Zeng, N, Li, W. Dihydroberberine alleviates Th17/Treg imbalance in premature ovarian insufficiency mice via inhibiting Rheb/mTOR signaling. Mol Med. 2024;30 (1):194. doi: 10.1186/s10020-024-00971-z. PubMed PMID:39472803 PubMed Central PMC11523677.
  14. Caferra, P, Fraisse, T, Trincavelli, ML, Marchetti, L, Piras, AM. Evaluation of orphan maintained biological medicinal products in the European Union between 2018 to 2023: a regulatory perspective. Expert Opin Biol Ther. 2024;24 (11):1279-1297. doi: 10.1080/14712598.2024.2422360. PubMed PMID:39460383 .
  15. Ruffenach, G, Le Ribeuz, H, Dutheil, M, El Jekmek, K, Dumont, F, Willer, AS et al.. Transcriptome analyses reveal common immune system dysregulation in PAH patients and Kcnk3-deficient rats. Pulm Circ. 2024;14 (4):e12434. doi: 10.1002/pul2.12434. PubMed PMID:39444497 PubMed Central PMC11497494.
  16. Twarda-Clapa, A. An update patent review of MDM2-p53 interaction inhibitors (2019-2023). Expert Opin Ther Pat. 2024;34 (12):1177-1198. doi: 10.1080/13543776.2024.2419836. PubMed PMID:39435470 .
  17. Chen, H, Xiang, Y, Tang, X, Hu, M. Establishment of a value assessment framework for orphan medicinal products in China. Orphanet J Rare Dis. 2024;19 (1):390. doi: 10.1186/s13023-024-03393-6. PubMed PMID:39428462 PubMed Central PMC11492536.
  18. Massey, S, Quigley, A, Rochfort, S, Christodoulou, J, Van Bergen, NJ. Cannabinoids and Genetic Epilepsy Models: A Review with Focus on CDKL5 Deficiency Disorder. Int J Mol Sci. 2024;25 (19):. doi: 10.3390/ijms251910768. PubMed PMID:39409097 PubMed Central PMC11476665.
  19. Loscocco, GG, Vannucchi, M, Santi, R, Amorosi, A, Scarpino, S, Siciliano, MC et al.. Hepatoid thymic carcinoma in a polycythemia vera patient treated with ropeginterferon Alfa-2b: Clinical, histopathological and molecular correlates. Pathol Res Pract. 2024;263 :155648. doi: 10.1016/j.prp.2024.155648. PubMed PMID:39405803 .
  20. Grabowski, H, DiMasi, JA, Long, G. Postapproval Innovation For Oncology Drugs And The Inflation Reduction Act. Health Aff (Millwood). 2024;43 (10):1400-1409. doi: 10.1377/hlthaff.2024.00202. PubMed PMID:39374451 .
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