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 .
Search PubMed