PUBLICATIONS

Publications on Orphan Drugs

  1. Kattner, AA. Ancient Wisdom and Modern Innovations: Methods of Administering Healing. Biomed J. 2024; :100773. doi: 10.1016/j.bj.2024.100773. PubMed PMID:39053731 .
  2. Little, RD, McKenzie, J, Srinivasan, A, Hilley, P, Gilmore, RB, Chee, D et al.. Switching from Dose-Intensified intravenous to SubCutaneoUS infliximab in Inflammatory Bowel Disease (DISCUS-IBD): protocol for a multicentre randomised controlled trial. BMJ Open. 2024;14 (7):e081787. doi: 10.1136/bmjopen-2023-081787. PubMed PMID:39032928 PubMed Central PMC11261670.
  3. Chen, Q, Xu, Y, Qu, R, Luo, X, Yang, Y. Clinical trial evidence supporting FDA approval of novel orphan drugs between 2017 and 2023. Drug Discov Today. 2024; :104102. doi: 10.1016/j.drudis.2024.104102. PubMed PMID:39032812 .
  4. Muenzer, J, Ho, C, Lau, H, Dant, M, Fuller, M, Boulos, N et al.. Community consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses. Mol Genet Metab. 2024;142 (4):108535. doi: 10.1016/j.ymgme.2024.108535. PubMed PMID:39018614 .
  5. Slominski, AT, Kim, TK, Janjetovic, Z, Slominski, RM, Li, W, Jetten, AM et al.. Biological Effects of CYP11A1-Derived Vitamin D and Lumisterol Metabolites in the Skin. J Invest Dermatol. 2024; :. doi: 10.1016/j.jid.2024.04.022. PubMed PMID:39001720 .
  6. El Saadany, T, Seitz, C, Bräm, C, Szucs, T. Bedside manufacturing as the next step in personalized medicine: medical progress and legal challenges. J Law Biosci. 2024;11 (2):lsae012. doi: 10.1093/jlb/lsae012. PubMed PMID:38989053 PubMed Central PMC11233777.
  7. Lakdawalla, DN, Phelps, CE, Nguyen, HH. Three Pillars of Support for Orphan Drug Programs: Individual Valuations, Societal Valuations, and Anonymous Altruism. Value Health. 2024; :. doi: 10.1016/j.jval.2024.06.014. PubMed PMID:38977180 .
  8. Bertsimas, D, Antonios Margonis, G, Sujichantararat, S, Koulouras, A, Ma, Y, Antonescu, CR et al.. Interpretable artificial intelligence to optimise use of imatinib after resection in patients with localised gastrointestinal stromal tumours: an observational cohort study. Lancet Oncol. 2024; :. doi: 10.1016/S1470-2045(24)00259-6. PubMed PMID:38976997 .
  9. Sinha, MS, Stern, AD, Rai, AK. Four Decades of Orphan Drugs and Priorities for the Future. N Engl J Med. 2024;391 (2):100-102. doi: 10.1056/NEJMp2401487. PubMed PMID:38973724 .
  10. Nieto-Gómez, P, Castaño-Amores, C. Factors influencing the reimbursement of cancer drugs in Europe: A scoping review. J Eval Clin Pract. 2024; :. doi: 10.1111/jep.14080. PubMed PMID:38959379 .
  11. Dienstman, J, Stachelek, SJ, Krieger, AM, Eskandanian, K, Espinoza, JC, Harrison, MR et al.. Pediatric Device Clinical Trials Activity: 1999-2022. Pediatrics. 2024; :. doi: 10.1542/peds.2023-063466. PubMed PMID:38953121 .
  12. Israr, J, Alam, S, Kumar, A. Drug repurposing for rare diseases. Prog Mol Biol Transl Sci. 2024;207 :231-247. doi: 10.1016/bs.pmbts.2024.03.034. PubMed PMID:38942540 .
  13. Liu, M, Lu, Y, Li, J, Zhang, Y, Zhang, S, Liu, Y et al.. Orphan drug policy analysis in China. Front Pharmacol. 2024;15 :1278710. doi: 10.3389/fphar.2024.1278710. PubMed PMID:38939834 PubMed Central PMC11208459.
  14. Eadie, AL, Simpson, JA, Brunt, KR. Teaching an old drug new tricks: Regulatory insights for the repurposing of hemin in cardiovascular disease. Pharmacol Res Perspect. 2024;12 (4):e1225. doi: 10.1002/prp2.1225. PubMed PMID:38923404 PubMed Central PMC11194834.
  15. Demirci, E, Knicley, J, Fiorentino, L. Clinical development and marketing application review times for novel orphan-designated drugs. Front Med (Lausanne). 2024;11 :1404922. doi: 10.3389/fmed.2024.1404922. PubMed PMID:38903827 PubMed Central PMC11188924.
  16. Mittal, A, Martin, MF, Levin, EJ, Adams, C, Yang, M, Provins, L et al.. Structures of synaptic vesicle protein 2A and 2B bound to anticonvulsants. Nat Struct Mol Biol. 2024; :. doi: 10.1038/s41594-024-01335-1. PubMed PMID:38898101 .
  17. Ng, QX, Ong, C, Chan, KE, Ong, TSK, Lim, IJX, Tang, ASP et al.. Comparative policy analysis of national rare disease funding policies in Australia, Singapore, South Korea, the United Kingdom and the United States: a scoping review. Health Econ Rev. 2024;14 (1):42. doi: 10.1186/s13561-024-00519-1. PubMed PMID:38896399 PubMed Central PMC11186122.
  18. Pottecher, J, Raffi, F, Jandrot-Perrus, M, Binay, S, Comenducci, A, Desort-Henin, V et al.. Targeting GPVI with glenzocimab in COVID-19 patients: Results from a randomized clinical trial. PLoS One. 2024;19 (6):e0302897. doi: 10.1371/journal.pone.0302897. PubMed PMID:38885234 PubMed Central PMC11182546.
  19. Alnaqbi, KA, Elshamy, AM, Gebran, N, Fahmy, S, Aldallal, S, Korra, N et al.. Consensus-Based Recommendations for the Implementation of Health Technology Assessment in the United Arab Emirates. Value Health Reg Issues. 2024;43 :101012. doi: 10.1016/j.vhri.2024.101012. PubMed PMID:38861786 .
  20. Stajszczyk, M, Batko, K, Żuber, ZM, Kwiatkowska, B, Krajewska-Włodarczyk, M, Batko, B et al.. Charting the Etanercept Journey: Tracing Cost Dynamics in Poland's Off-Patent Market from Reference Drug Rivalry to Biosimilar Monopoly. BioDrugs. 2024;38 (4):557-569. doi: 10.1007/s40259-024-00663-4. PubMed PMID:38861154 PubMed Central PMC11247056.
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