Publications on Orphan Drugs

  1. 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.
  2. 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 .
  3. 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.
  4. 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.
  5. 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 .
  6. Shin, EY, Hong, YJ, Lim, KM, Kim, TH, Lee, JH. International comparison of availability for orphan drugs: focused on approved orphan drugs in South Korea. J Pharm Policy Pract. 2024;17 (1):2354299. doi: 10.1080/20523211.2024.2354299. PubMed PMID:38845625 PubMed Central PMC11155423.
  7. Birgül Iyison, N, Abboud, C, Abboud, D, Abdulrahman, AO, Bondar, AN, Dam, J et al.. ERNEST COST action overview on the (patho)physiology of GPCRs and orphan GPCRs in the nervous system. Br J Pharmacol. 2024; :. doi: 10.1111/bph.16389. PubMed PMID:38825750 .
  8. Fasseeh, AN, Korra, N, Elezbawy, B, Sedrak, AS, Gamal, M, Eldessouki, R et al.. Framework for developing cost-effectiveness analysis threshold: the case of Egypt. J Egypt Public Health Assoc. 2024;99 (1):12. doi: 10.1186/s42506-024-00159-7. PubMed PMID:38825614 PubMed Central PMC11144683.
  9. Kato, A, Iwasaki, R, Takeshima, K, Maruzuru, Y, Koyanagi, N, Natsume, T et al.. Identification of a novel neurovirulence factor encoded by the cryptic orphan gene UL31.6 of herpes simplex virus 1. J Virol. 2024; :e0074724. doi: 10.1128/jvi.00747-24. PubMed PMID:38819171 .
  10. Bretos-Azcona, PE, Wallace, M, Jootun, M, Jin, G, Agirrezabal, I, Szende, A et al.. An Early Cost-Utility Model of mRNA-Based Therapies for the Treatment of Methylmalonic and Propionic Acidemia in the United Kingdom. Clin Drug Investig. 2024;44 (6):399-412. doi: 10.1007/s40261-024-01363-1. PubMed PMID:38796677 .
  11. Alonso Ruiz, A, Large, K, Moon, S, Vieira, M. Pharmaceutical policy and innovation for rare diseases: A narrative review. F1000Res. 2023;12 :211. doi: 10.12688/f1000research.130809.2. PubMed PMID:38778810 PubMed Central PMC11109548.
  12. Santos, JV, Padron-Monedero, A, Bikbov, B, Grad, DA, Plass, D, Mechili, EA et al.. The state of health in the European Union (EU-27) in 2019: a systematic analysis for the Global Burden of Disease study 2019. BMC Public Health. 2024;24 (1):1374. doi: 10.1186/s12889-024-18529-3. PubMed PMID:38778362 PubMed Central PMC11110444.
  13. Zhang, S, Hu, Y, Zhao, Y, Feng, Y, Wang, X, Miao, M et al.. Molecular mechanism of Chang Shen Hua volatile oil modulating brain cAMP-PKA-CREB pathway to improve depression-like behavior in rats. Phytomedicine. 2024;130 :155729. doi: 10.1016/j.phymed.2024.155729. PubMed PMID:38772184 .
  14. Sampei, C, Kato, K, Arasaki, Y, Kimura, Y, Konno, T, Otsuka, K et al.. Gprc5a is a novel parathyroid hormone-inducible gene and negatively regulates osteoblast proliferation and differentiation. J Cell Physiol. 2024; :. doi: 10.1002/jcp.31297. PubMed PMID:38769895 .
  15. GBD 2021 Risk Factors Collaborators. Global burden and strength of evidence for 88 risk factors in 204 countries and 811 subnational locations, 1990-2021: a systematic analysis for the Global Burden of Disease Study 2021. Lancet. 2024;403 (10440):2162-2203. doi: 10.1016/S0140-6736(24)00933-4. PubMed PMID:38762324 PubMed Central PMC11120204.
  16. Kediha, MI, Tazir, M, Sternberg, D, Eymard, B, Ali Pacha, L. Innovative Therapeutic Approaches in Congenital Myasthenic Syndromes. Neurol Clin Pract. 2024;14 (3):e200277. doi: 10.1212/CPJ.0000000000200277. PubMed PMID:38737513 PubMed Central PMC11081764.
  17. Felippini, A, Biglia, LV, Lima, TM, Aguiar, PM. HTA criteria adopted in different models of public healthcare systems for orphan drugs: A scoping review. Health Policy. 2024;144 :105080. doi: 10.1016/j.healthpol.2024.105080. PubMed PMID:38733643 .
  18. Erceg, D, Jakirović, M, Prgomet, L, Madunić, M, Turkalj, M. Conducting Drug Treatment Trials in Children: Opportunities and Challenges. Pharmaceut Med. 2024;38 (3):179-204. doi: 10.1007/s40290-024-00523-0. PubMed PMID:38730200 .
  19. Hu, Y, Pang, Z. A novel MCGDM technique based on correlation coefficients under probabilistic hesitant fuzzy environment and its application in clinical comprehensive evaluation of orphan drugs. PLoS One. 2024;19 (5):e0303042. doi: 10.1371/journal.pone.0303042. PubMed PMID:38709744 PubMed Central PMC11073718.
  20. Nicholls, SJ, Nelson, AJ, Ditmarsch, M, Kastelein, JJP, Ballantyne, CM, Ray, KK et al.. Obicetrapib on top of maximally tolerated lipid-modifying therapies in participants with or at high risk for atherosclerotic cardiovascular disease: rationale and designs of BROADWAY and BROOKLYN. Am Heart J. 2024;274 :32-45. doi: 10.1016/j.ahj.2024.05.002. PubMed PMID:38705341 .
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