PUBLICATIONS

Publications on Orphan Drugs

  1. 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; :. doi: 10.1021/acs.jmedchem.4c01727. PubMed PMID:39546350 .
  2. 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 .
  3. 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 .
  4. 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.
  5. 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.
  6. 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.
  7. 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 .
  8. 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.
  9. Coats, JT, Li, S, Tanaka, TU, Tauro, S, Sutherland, C, Saurin, AT et al.. Elraglusib induces cytotoxicity via direct microtubule destabilization independently of GSK3 inhibition. Cancer Res Commun. 2024; :. doi: 10.1158/2767-9764.CRC-24-0408. PubMed PMID:39470360 .
  10. 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; :1-19. doi: 10.1080/14712598.2024.2422360. PubMed PMID:39460383 .
  11. 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.
  12. Twarda-Clapa, A. An update patent review of MDM2-p53 interaction inhibitors (2019-2023). Expert Opin Ther Pat. 2024; :. doi: 10.1080/13543776.2024.2419836. PubMed PMID:39435470 .
  13. 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.
  14. 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.
  15. 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 .
  16. 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 .
  17. Badrane, I, Urso, L, Campennì, A, Cittanti, C, De Rimini, ML, Bartolomei, M et al.. Radioligand therapy in sympathetic-adrenal-medullary axis tumors: state of art and perspectives. Endocrine. 2024; :. doi: 10.1007/s12020-024-04062-1. PubMed PMID:39373830 .
  18. Uwitonze, JP, Duminy, L, Blankart, CR. Identifying health inequities faced by older adults with rare diseases: A systematic literature review and proposal for an ethical spectrum and resource allocation framework. Health Policy. 2024;149 :105176. doi: 10.1016/j.healthpol.2024.105176. PubMed PMID:39348734 .
  19. do Nascimento, RRNR, Piotto, DGP, Freire, EAM, de Souza Neves, F, Sztajnbok, FR, Bica, BERG et al.. Rare diseases: What rheumatologists need to know?. Adv Rheumatol. 2024;64 (1):74. doi: 10.1186/s42358-024-00407-6. PubMed PMID:39334496 .
  20. Cai, Y, Sui, L, Wang, J, Qian, W, Peng, Y, Gong, L et al.. Post-marketing surveillance framework of cell and gene therapy products in the European Union, the United States, Japan, South Korea and China: a comparative study. BMC Med. 2024;22 (1):421. doi: 10.1186/s12916-024-03637-z. PubMed PMID:39334246 PubMed Central PMC11438358.
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