PUBLICATIONS

Publications on Orphan Drugs

  1. Frost, J, Widening Engagement Patient Advisory Group, Mandizha, J, van den Dungen, C, Asare, L, Pope, C et al.. Can the Development of Orphan Drugs Include Wider Patient Engagement? A Citizens' Jury to Explore a Promissory Notion. Health Expect. 2025;28 (6):e70524. doi: 10.1111/hex.70524. PubMed PMID:41347535 PubMed Central PMC12679173.
  2. Parker, S, Hajjar, JE, Jonker, AH, Kahn, SR, Kritikou, P, Kyryiakopoulou, C et al.. Non-oncology orphan drug development: Productivity and probability of success. Drug Discov Today. 2025; :104568. doi: 10.1016/j.drudis.2025.104568. PubMed PMID:41344392 .
  3. Oliveira, BA, Torres, A, Ricci-Júnior, E, Almeida, IF, Monteiro, MSSB. Topical Treatments for Rare Genetic Dermatological Diseases: A Narrative Review. Pharmaceuticals (Basel). 2025;18 (11):. doi: 10.3390/ph18111762. PubMed PMID:41305004 PubMed Central PMC12655290.
  4. Hu, C, Dong, J, Yuan, J, Shang, S, Zhai, X, Wen, Y et al.. Effect of Brassinolide on the Growth and Physiological Indicators of Foxtail Millet Under Cyhalofop-Butyl Damage. Plants (Basel). 2025;14 (22):. doi: 10.3390/plants14223421. PubMed PMID:41304572 PubMed Central PMC12656274.
  5. Marcellusi, A, Cazzato, D, Guarnotta, G, Aiello, A, Bonfanti, M, Bitonti, R et al.. Horizon scanning and drug expenditure for rare diseases: three-year predictive model in Italy 2025-2027. Health Econ Rev. 2025; :. doi: 10.1186/s13561-025-00699-4. PubMed PMID:41296238 .
  6. Grynblat, J, Hlavaty, A, Savale, L, Weatherald, J, Le Bozec, A, Aguado, B et al.. Drugs and toxins associated with pulmonary arterial hypertension: from established culprits to novel threats. ERJ Open Res. 2025;11 (6):. doi: 10.1183/23120541.00382-2025. PubMed PMID:41293777 PubMed Central PMC12643032.
  7. Mooney, H, Kesselheim, AS, Rome, BN. US spending on high-revenue rare disease drugs in 2022. Am J Manag Care. 2025;31 (11):e329-e335. doi: 10.37765/ajmc.2025.89823. PubMed PMID:41289291 .
  8. Pan, I, Lin, C, Palestine, AG, Edwards Mayhew, RG, Jung, JL. Use of Tacrolimus for the Treatment of Pediatric Noninfectious Uveitis. Ophthalmol Ther. 2025; :. doi: 10.1007/s40123-025-01275-z. PubMed PMID:41275437 .
  9. Kumar, A, Shukla, S, Rai, A, Pathak, P, Narayan, KP. Concurrent nanotherapeutics and regulatory updates for the management of amyotrophic lateral sclerosis: a focused review for orphan drug (Tofersen). Orphanet J Rare Dis. 2025;20 (1):598. doi: 10.1186/s13023-025-04042-2. PubMed PMID:41272780 PubMed Central PMC12639972.
  10. Conduit, SE, Zhang, CXW, Pearce, W, Guillermet-Guibert, J, Sferruzzi-Perri, AN, Vanhaesebroeck, B et al.. Novel role for PI3Kβ in placental function through regulation of system A amino acid transporter expression, associated with embryonic lethality. Cell Mol Life Sci. 2025;82 (1):413. doi: 10.1007/s00018-025-05937-w. PubMed PMID:41258474 PubMed Central PMC12630484.
  11. Rosenthal, GL, Peiris, V, Torjusen, E, Federici, T, Schwartz, SB, Ricci, LJ et al.. A national strategic framework for developing medical devices for children. Pediatr Res. 2025; :. doi: 10.1038/s41390-025-04548-2. PubMed PMID:41254176 .
  12. Benchetrit, S, Chu, C, Athanasiou, D, Scherer, S, de Andres Trelles, F, Stoyanova-Beninska, V et al.. Paediatric medicines development for Duchenne muscular dystrophy: An EU regulatory perspective. J Neuromuscul Dis. 2025; :22143602251397736. doi: 10.1177/22143602251397736. PubMed PMID:41252306 .
  13. Versteeg, JW, Stam, N, Mantel-Teeuwisse, AK, Timmers, L, Goettsch, W, Leopold, C et al.. Coverage with evidence development for medicines with insufficient evidence of clinical benefit: experience from the Netherlands. Int J Technol Assess Health Care. 2025;41 (1):e83. doi: 10.1017/S0266462325103267. PubMed PMID:41243444 .
  14. Zarbock, A, Ostermann, M, Forni, L, Bode, C, Wild, L, Putensen, C et al.. A preventive care strategy to reduce moderate or severe acute kidney injury after major surgery (BigpAK-2); a multinational, randomised clinical trial. Lancet. 2025; :. doi: 10.1016/S0140-6736(25)01717-9. PubMed PMID:41242333 .
  15. Komarov, IA. [THE ISSUES OF MEDICINAL SUPPORT UNDER HIGHLY WASTEFUL NOSOLOGIES. THE ORIGINAL MEDICINAL PREPARATIONS REPRODUCED ONES AND BIO-ANALOGUES]. Probl Sotsialnoi Gig Zdravookhranenniiai Istor Med. 2025;33 (5):1230-1235. doi: 10.32687/0869-866X-2025-33-5-1230-1235. PubMed PMID:41236316 .
  16. Cousin, EM, Martin, K, Hansen, RN, Coster, J, Sullivan, SD. Drugs anticipated to be selected for Medicare price negotiation in 2026 for implementation in 2028. J Manag Care Spec Pharm. 2025; :1-11. doi: 10.18553/jmcp.2025.25253. PubMed PMID:41235761 .
  17. Rosenberg, N, Post, HC, Schutte, T, de Visser, SJ, Bartelink, I, Pasmooij, AMG et al.. Access to anticancer and orphan medicines through compassionate use programs and named patient basis in seven European countries. ESMO Open. 2025;10 (11):105855. doi: 10.1016/j.esmoop.2025.105855. PubMed PMID:41232200 PubMed Central PMC12661435.
  18. Hu, C, Chen, T, Diao, C, Dou, B, Shang, S, Li, S et al.. Regulatory Effects of Different Doses of Penoxsulam on Endogenous Hormones and Antioxidant System in Foxtail Millet. Plants (Basel). 2025;14 (21):. doi: 10.3390/plants14213254. PubMed PMID:41225806 PubMed Central PMC12610494.
  19. Hu, S, Li, X, Yu, M, Zhang, H, Wang, X, Qi, C et al.. Design, synthesis, and evaluation of 5-((4-(pyridin-3-yl)pyrimidin-2-yl)amino)-1H-indole-2-carboxamide derivatives as anti-HCC agents targeting Nur77. Bioorg Chem. 2025;167 :109113. doi: 10.1016/j.bioorg.2025.109113. PubMed PMID:41223584 .
  20. Lexchin, J. Delay in the Submission of the Results of Confirmatory Studies to Health Canada: A Cross-Sectional Study. J Gen Intern Med. 2025; :. doi: 10.1007/s11606-025-10040-8. PubMed PMID:41217641 .
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