PUBLICATIONS

Publications on Orphan Drugs

  1. Margelidon-Cozzolino, V, Balsamelli, J, Carrard, J, Ait Yahia, S, Gevaert, MH, Demoulin-Alexikova, S et al.. Dog allergen-induced asthma in mice: a relevant model of T2low severe asthma with airway remodelling. Inflamm Res. 2025;74 (1):52. doi: 10.1007/s00011-025-02004-9. PubMed PMID:40082266 .
  2. Tafuri, G, Bracco, A, Grueger, J. Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective. A 2025 update. Expert Rev Pharmacoecon Outcomes Res. 2025; :. doi: 10.1080/14737167.2025.2479131. PubMed PMID:40077941 .
  3. Hanchard, MS. Debates over orphan drug pricing: a meta-narrative literature review. Orphanet J Rare Dis. 2025;20 (1):107. doi: 10.1186/s13023-025-03634-2. PubMed PMID:40055799 PubMed Central PMC11887186.
  4. Jaksa, A, Arena, PJ, Hanisch, M, Marsico, M. Use of Real-World Evidence in Health Technology Reassessments Across Six Health Technology Assessment Agencies. Value Health. 2025; :. doi: 10.1016/j.jval.2025.02.012. PubMed PMID:40054770 .
  5. GBD 2021 Adult BMI Collaborators. Global, regional, and national prevalence of adult overweight and obesity, 1990-2021, with forecasts to 2050: a forecasting study for the Global Burden of Disease Study 2021. Lancet. 2025;405 (10481):813-838. doi: 10.1016/S0140-6736(25)00355-1. PubMed PMID:40049186 .
  6. GBD 2021 Adolescent BMI Collaborators. Global, regional, and national prevalence of child and adolescent overweight and obesity, 1990-2021, with forecasts to 2050: a forecasting study for the Global Burden of Disease Study 2021. Lancet. 2025;405 (10481):785-812. doi: 10.1016/S0140-6736(25)00397-6. PubMed PMID:40049185 .
  7. Kong, Q, Fan, C, Zhang, Y, Yan, X, Chen, L, Kang, Q et al.. Rare disease publishing trends worldwide and in China: A CiteSpace-based bibliometric study. Intractable Rare Dis Res. 2025;14 (1):1-13. doi: 10.5582/irdr.2024.01059. PubMed PMID:40046029 PubMed Central PMC11878222.
  8. Enright, DE, van Duijnhoven, EG, Ollendorf, DA, Chambers, JD. Use of health technology assessments in specialty drug coverage decisions by US commercial health plans. J Manag Care Spec Pharm. 2025;31 (3):289-295. doi: 10.18553/jmcp.2025.31.3.289. PubMed PMID:40021468 PubMed Central PMC11871157.
  9. Howell, KB, White, SM, McTague, A, D'Gama, AM, Costain, G, Poduri, A et al.. International Precision Child Health Partnership (IPCHiP): an initiative to accelerate discovery and improve outcomes in rare pediatric disease. NPJ Genom Med. 2025;10 (1):13. doi: 10.1038/s41525-025-00474-8. PubMed PMID:40016282 PubMed Central PMC11868529.
  10. Fasseeh, AN, Korra, N, Aljedai, A, Seyam, A, Almudaiheem, H, Al-Abdulkarim, HA et al.. Rare disease challenges and potential actions in the Middle East. Int J Equity Health. 2025;24 (1):56. doi: 10.1186/s12939-025-02388-4. PubMed PMID:40011905 PubMed Central PMC11863865.
  11. Murakami, C, Atsuta-Tsunoda, K, Inomata, S, Kawai, T, Hijikata, Y, Dilimulati, K et al.. Human PHOSPHO1 exhibits phosphatidylcholine- and phosphatidylethanolamine-phospholipase C activities and interacts with diacylglycerol kinase δ. FEBS Lett. 2025; :. doi: 10.1002/1873-3468.70018. PubMed PMID:39992810 .
  12. Rahaghi, FF, Humbert, M, Hoeper, MM, White, RJ, Frantz, RP, Hassoun, PM et al.. Future treatment paradigms in pulmonary arterial hypertension: a personal view from physicians, health authorities, and patients. Lancet Respir Med. 2025; :. doi: 10.1016/S2213-2600(24)00425-9. PubMed PMID:39987941 .
  13. GBD 2021 Europe Life Expectancy Collaborators. Changing life expectancy in European countries 1990-2021: a subanalysis of causes and risk factors from the Global Burden of Disease Study 2021. Lancet Public Health. 2025;10 (3):e172-e188. doi: 10.1016/S2468-2667(25)00009-X. PubMed PMID:39983748 PubMed Central PMC11876102.
  14. Windecker, S, Fraser, AG, Szymanski, P, Gilard, M, Lüscher, TF, Abid, L et al.. Priorities for medical device regulatory approval: a report from the European Society of Cardiology Cardiovascular Round Table. Eur Heart J. 2025; :. doi: 10.1093/eurheartj/ehaf069. PubMed PMID:39977258 .
  15. Topouzis, S, Papapetropoulos, A, Alexander, SPH, Cortese-Krott, M, Kendall, DA, Martemyanov, K et al.. Novel drugs approved by the EMA, the FDA and the MHRA in 2024: A year in review. Br J Pharmacol. 2025;182 (7):1416-1445. doi: 10.1111/bph.17458. PubMed PMID:39971274 .
  16. Cacoub, E, Lefebvre, NB, Milunov, D, Sarkar, M, Saha, S. Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics. Front Public Health. 2025;13 :1520467. doi: 10.3389/fpubh.2025.1520467. PubMed PMID:39963479 PubMed Central PMC11830808.
  17. Yuanyuan, AN, Wang, L, Yanjie, LI, Yanchun, W, Xiaobin, R, Hongbing, HE et al.. Effect and mechanism of Sanqi in treating periodontitis. J Tradit Chin Med. 2025;45 (1):66-75. doi: 10.19852/j.cnki.jtcm.20241111.001. PubMed PMID:39957160 PubMed Central PMC11764944.
  18. Louie, AD, Huntington, KE, Lee, Y, Mompoint, J, Wu, LJ, Lee, S et al.. TRAIL receptor agonist TLY012 in combination with PD-1 inhibition promotes tumor regression in an immune-competent mouse model of pancreatic ductal adenocarcinoma. Am J Cancer Res. 2025;15 (1):286-298. doi: 10.62347/ROAT5658. PubMed PMID:39949937 PubMed Central PMC11815385.
  19. Kummar, S, Bui, N, Messersmith, WA, Whiting, J, Portnoy, M, Lim, A et al.. Nirogacestat-the pathway to approval of the first treatment for desmoid tumors, a rare disease. Ther Adv Rare Dis. 2025;6 :26330040251317546. doi: 10.1177/26330040251317546. PubMed PMID:39944188 PubMed Central PMC11815814.
  20. Ozeri, DJ, Bar, D, Somech Safran, B, Druyan, A, Kukuy, OL, Giat, E et al.. Renal outcomes and survival in amyloidosis associated with familial Mediterranean fever: A longitudinal study. Semin Arthritis Rheum. 2025;71 :152642. doi: 10.1016/j.semarthrit.2025.152642. PubMed PMID:39908750 .
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