Publications on Orphan Drugs

  1. Guerrini, R, Chiron, C, Vandame, D, Linley, W, Toward, T. Comparative efficacy and safety of stiripentol, cannabidiol and fenfluramine as first-line add-on therapies for seizures in Dravet syndrome: A network meta-analysis. Epilepsia Open. 2024; :. doi: 10.1002/epi4.12923. PubMed PMID:38427284 .
  2. Bouwman, L, Sepodes, B, Leufkens, H, Torre, C. Trends in orphan medicinal products approvals in the European Union between 2010-2022. Orphanet J Rare Dis. 2024;19 (1):91. doi: 10.1186/s13023-024-03095-z. PubMed PMID:38413985 PubMed Central PMC10900541.
  3. Mease, C, Miller, KL, Fermaglich, LJ, Best, J, Liu, G, Torjusen, E et al.. Analysis of the first ten years of FDA's rare pediatric disease priority review voucher program: designations, diseases, and drug development. Orphanet J Rare Dis. 2024;19 (1):86. doi: 10.1186/s13023-024-03097-x. PubMed PMID:38403586 PubMed Central PMC10895788.
  4. Chirmule, N, Feng, H, Cyril, E, Ghalsasi, VV, Choudhury, MC. Orphan drug development: Challenges, regulation, and success stories. J Biosci. 2024;49 :. . PubMed PMID:38383975 .
  5. Clemente, T, Galli, L, Lolatto, R, Gagliardini, R, Lagi, F, Ferrara, M et al.. Cohort profile: PRESTIGIO, an Italian prospective registry-based cohort of people with HIV-1 resistant to reverse transcriptase, protease and integrase inhibitors. BMJ Open. 2024;14 (2):e080606. doi: 10.1136/bmjopen-2023-080606. PubMed PMID:38341206 PubMed Central PMC10862296.
  6. Csorba, A, Katona, G, Budai-Szűcs, M, Balogh-Weiser, D, Molnár, P, Maka, E et al.. A Comparative Pharmacokinetic Study for Cysteamine-Containing Eye Drops as an Orphan Topical Therapy in Cystinosis. Int J Mol Sci. 2024;25 (3):. doi: 10.3390/ijms25031623. PubMed PMID:38338900 PubMed Central PMC10855117.
  7. Seoane-Vazquez, E, Rodriguez-Monguio, R, Powers, JH 3rd. Analysis of US Food and Drug Administration new drug and biologic approvals, regulatory pathways, and review times, 1980-2022. Sci Rep. 2024;14 (1):3325. doi: 10.1038/s41598-024-53554-7. PubMed PMID:38336899 PubMed Central PMC10858128.
  8. Santoro, JD, Sico, JJ, Burke, JF, Sarkar, K, Turbes, M, Evans, DA et al.. Prescription Drug Prices: An AAN Position Statement. Neurology. 2024;102 (5):e209132. doi: 10.1212/WNL.0000000000209132. PubMed PMID:38335469 .
  9. Lexchin, J. Therapeutic Value of Orphan Drugs Approved by Health Canada: A Cross-Sectional Study. J Gen Intern Med. 2024; :. doi: 10.1007/s11606-024-08651-8. PubMed PMID:38326584 .
  10. Seror, R, Chiche, L, Beydon, M, Desjeux, G, Zhuo, J, Vannier-Moreau, V et al.. Estimated prevalence, incidence and healthcare costs of Sjögren's syndrome in France: a national claims-based study. RMD Open. 2024;10 (1):. doi: 10.1136/rmdopen-2023-003591. PubMed PMID:38307699 PubMed Central PMC10840052.
  11. Al-Jedai, A, Almudaiheem, H, Alruthia, Y, Althemery, A, Alabdulkarim, H, Ojeil, R et al.. A Step Toward the Development of the First National Multi-Criteria Decision Analysis Framework to Support Healthcare Decision Making in Saudi Arabia. Value Health Reg Issues. 2024;41 :100-107. doi: 10.1016/j.vhri.2023.12.005. PubMed PMID:38306770 .
  12. Coleman, M, Mascialino, SJ, Panjwani, A, Edwards, E, Sukhatme, VV, Gavegnano, C et al.. Readily available drugs and other interventions to potentially improve the efficacy of immune checkpoint blockade in cancer. Front Immunol. 2023;14 :1281744. doi: 10.3389/fimmu.2023.1281744. PubMed PMID:38299150 PubMed Central PMC10827885.
  13. Jonker, AH, O'Connor, D, Cavaller-Bellaubi, M, Fetro, C, Gogou, M, 't Hoen, PAC et al.. Drug repurposing for rare: progress and opportunities for the rare disease community. Front Med (Lausanne). 2024;11 :1352803. doi: 10.3389/fmed.2024.1352803. PubMed PMID:38298814 PubMed Central PMC10828010.
  14. Zanardi, A, Nardini, I, Raia, S, Conti, A, Ferrini, B, D'Adamo, P et al.. New orphan disease therapies from the proteome of industrial plasma processing waste- a treatment for aceruloplasminemia. Commun Biol. 2024;7 (1):140. doi: 10.1038/s42003-024-05820-7. PubMed PMID:38291108 PubMed Central PMC10828504.
  15. Chen, G, Staffen, N, Wu, Z, Xu, X, Pan, J, Inoue, A et al.. Structural and functional characterization of the endogenous agonist for orphan receptor GPR3. Cell Res. 2024;34 (3):262-265. doi: 10.1038/s41422-023-00919-8. PubMed PMID:38287118 PubMed Central PMC10907609.
  16. Reilmann, R, Anderson, KE, Feigin, A, Tabrizi, SJ, Leavitt, BR, Stout, JC et al.. Safety and efficacy of laquinimod for Huntington's disease (LEGATO-HD): a multicentre, randomised, double-blind, placebo-controlled, phase 2 study. Lancet Neurol. 2024;23 (3):243-255. doi: 10.1016/S1474-4422(23)00454-4. PubMed PMID:38280392 .
  17. Belot, A, Benezech, S, Tusseau, M. A new drug for rare diseases: pozelimab for CHAPLE disease. Lancet. 2024;403 (10427):592-593. doi: 10.1016/S0140-6736(23)02652-1. PubMed PMID:38278169 .
  18. Yang, JP, Leadman, D, Ballew, RM, Sid, E, Xu, Y, Mathé, EA et al.. User Centered Rare Disease Clinical Trial Knowledge Graph (RCTKG). Stud Health Technol Inform. 2024;310 :94-98. doi: 10.3233/SHTI230934. PubMed PMID:38269772 .
  19. Zhu, X, Chen, Y. Bridging the new drug access gap between China and the United States and its related policies. Front Pharmacol. 2023;14 :1296737. doi: 10.3389/fphar.2023.1296737. PubMed PMID:38259295 PubMed Central PMC10800674.
  20. Russell, MD, Massey, J, Roddy, E, MacKenna, B, Bacon, S, Goldacre, B et al.. Gout incidence and management during the COVID-19 pandemic in England, UK: a nationwide observational study using OpenSAFELY. Lancet Rheumatol. 2023;5 (10):e622-e632. doi: 10.1016/S2665-9913(23)00206-0. PubMed PMID:38251486 .
Search PubMed