PUBLICATIONS

Publications on Orphan Drugs

  1. Weatherald, J, Boucly, A, Peters, A, Montani, D, Prasad, K, Psotka, MA et al.. The evolving landscape of pulmonary arterial hypertension clinical trials. Lancet. 2022;400 (10366):1884-1898. doi: 10.1016/S0140-6736(22)01601-4. PubMed PMID:36436527 .
  2. Roberts, SW, Elvang, TLB, Syed, L, Samuelsen, MB, Arp-Hansen, EL, Nielsen, HK et al.. Regulatory Considerations Toward Orphan Drug Designation and Orphan Drug Exclusivity in the United States and European Union: Structural Similarity, Clinical Superiority/Significant Benefit, and Case Studies. Ther Innov Regul Sci. 2022; :. doi: 10.1007/s43441-022-00477-y. PubMed PMID:36401076 .
  3. Christiansen, H, De Bruin, ML, Hallgreen, CE. Mandatory requirements for pediatric drug development in the EU and the US for novel drugs-A comparative study. Front Med (Lausanne). 2022;9 :1009432. doi: 10.3389/fmed.2022.1009432. PubMed PMID:36388912 PubMed Central PMC9659631.
  4. Kawakami, A, Masamune, K. The actual status of drug prices and adjustment factors for drug price calculation: an analysis of ultra-orphan drug development in Japan. Orphanet J Rare Dis. 2022;17 (1):408. doi: 10.1186/s13023-022-02526-z. PubMed PMID:36348359 PubMed Central PMC9641848.
  5. Nakao, M, Nakamura, Y, Shimokawa, M, Maeda, H. Postmarketing all-case surveillance trends and contribution to safety measures of drugs approved in Japan: a cross-sectional survey in 1999-2019. Int J Clin Pharm. 2022; :. doi: 10.1007/s11096-022-01461-0. PubMed PMID:36323961 .
  6. Han, Q, Fu, H, Chu, X, Wen, R, Zhang, M, You, T et al.. Research advances in treatment methods and drug development for rare diseases. Front Pharmacol. 2022;13 :971541. doi: 10.3389/fphar.2022.971541. PubMed PMID:36313320 PubMed Central PMC9597619.
  7. Denton, N, Mulberg, AE, Molloy, M, Charleston, S, Fajgenbaum, DC, Marsh, ED et al.. Sharing is caring: a call for a new era of rare disease research and development. Orphanet J Rare Dis. 2022;17 (1):389. doi: 10.1186/s13023-022-02529-w. PubMed PMID:36303170 PubMed Central PMC9612604.
  8. García-Parra, B, Guiu, JM, Modamio, P, Martínez-Yélamos, A, Mariño-Hernández, EL, Povedano, M et al.. [Access to orphan drugs for the treatment of spinal muscular atrophy in Spain]. Rev Neurol. 2022;75 (9):261-267. doi: 10.33588/rn.7509.2022298. PubMed PMID:36285446 .
  9. Saydam, M, Timur, SS, Vural, İ, Takka, S. Cell culture and pharmacokinetic evaluation of a solid dosage formulation containing a water-insoluble orphan drug manufactured by FDM-3DP technology. Int J Pharm. 2022;628 :122307. doi: 10.1016/j.ijpharm.2022.122307. PubMed PMID:36270551 .
  10. Pizzamiglio, C, Vernon, HJ, Hanna, MG, Pitceathly, RDS. Designing clinical trials for rare diseases: unique challenges and opportunities. Nat Rev Methods Primers. 2022;2 (1):. doi: 10.1038/s43586-022-00100-2. PubMed PMID:36254116 PubMed Central PMC7613711.
  11. Guennif, S. Capture and passive predation in times of COVID-19 pandemic. Public Choice. 2022;193 (3-4):163-186. doi: 10.1007/s11127-022-01005-0. PubMed PMID:36248699 PubMed Central PMC9547627.
  12. Żelewski, P, Wojna, M, Sygit, K, Cipora, E, Gąska, I, Niemiec, M et al.. Comparison of US and EU Prices for Orphan Drugs in the Perspective of the Considered US Orphan Drugs Act Modifications and Discussed Price-Regulation Mechanisms Adjustments in US and European Union. Int J Environ Res Public Health. 2022;19 (19):. doi: 10.3390/ijerph191912098. PubMed PMID:36231399 PubMed Central PMC9566473.
  13. Martin, PA. The challenge of institutionalised complicity: Researching the pharmaceutical industry in the era of impact and engagement. Sociol Health Illn. 2022; :. doi: 10.1111/1467-9566.13536. PubMed PMID:36217290 .
  14. Lu, Y, Gao, Q, Ren, X, Li, J, Yang, D, Zhang, Z et al.. Incidence and prevalence of 121 rare diseases in China: Current status and challenges: 2022 revision. Intractable Rare Dis Res. 2022;11 (3):96-104. doi: 10.5582/irdr.2022.01093. PubMed PMID:36200031 PubMed Central PMC9438002.
  15. Has, C, Hess, M, Anemüller, W, Blume-Peytavi, U, Emmert, S, Fölster-Holst, R et al.. Epidemiology of inherited epidermolysis bullosa in Germany. J Eur Acad Dermatol Venereol. 2022; :. doi: 10.1111/jdv.18637. PubMed PMID:36196047 .
  16. Shengnan, D, Zixuan, L, Na, Z, Weikai, Z, Yuanyuan, Y, Jiasu, L et al.. Using 5 consecutive years of NICE guidance to describe the characteristics and influencing factors on the economic evaluation of orphan oncology drugs. Front Public Health. 2022;10 :964040. doi: 10.3389/fpubh.2022.964040. PubMed PMID:36187695 PubMed Central PMC9519130.
  17. Rahman, MM, Islam, MR, Mim, SA, Sultana, N, Chellappan, DK, Dua, K et al.. Insights into the Promising Prospect of G Protein and GPCR-Mediated Signaling in Neuropathophysiology and Its Therapeutic Regulation. Oxid Med Cell Longev. 2022;2022 :8425640. doi: 10.1155/2022/8425640. PubMed PMID:36187336 PubMed Central PMC9519337.
  18. Chen, X, Tu, X, Zhang, X, Cao, B, Liu, W, Zhang, J et al.. SAR study of oxidative DIMs analogs targeting the Nur77-mediated apoptotic pathway of cancer cells. Bioorg Chem. 2022;129 :106156. doi: 10.1016/j.bioorg.2022.106156. PubMed PMID:36179441 .
  19. Siepe, DH, Henneberg, LT, Wilson, SC, Hess, GT, Bassik, MC, Zinn, K et al.. Identification of orphan ligand-receptor relationships using a cell-based CRISPRa enrichment screening platform. Elife. 2022;11 :. doi: 10.7554/eLife.81398. PubMed PMID:36178190 PubMed Central PMC9578707.
  20. Bösch, J, Rugg, C, Schäfer, V, Lichtenberger, P, Staier, N, Treichl, B et al.. Low-Molecular-Weight Heparin Resistance and Its Viscoelastic Assessment in Critically Ill COVID-19 Patients. Semin Thromb Hemost. 2022;48 (7):850-857. doi: 10.1055/s-0042-1756304. PubMed PMID:36174602 .
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