PUBLICATIONS

Publications on Orphan Drugs

  1. Mugerwa, M, Namutundu, J, Nangendo, J, Babirye Tumusiime, V, Ndekezi, D, Atuheire, CGK et al.. Disclosure of positive HIV status to sexual partners among young people receiving treatment at an urban clinic, Kampala, Uganda. AIDS Res Ther. 2025;22 (1):40. doi: 10.1186/s12981-025-00727-7. PubMed PMID:40170187 .
  2. Agavriloaei, BD, Costache, RC, Babici, RG, Racoviță, S, Dodi, G, Covic, AC et al.. The prospect of novel orphan therapeutic protocol for TSC2/PKD1 contiguous gene syndrome: a case report. BMC Nephrol. 2025;26 (1):166. doi: 10.1186/s12882-025-04088-1. PubMed PMID:40170159 .
  3. Costa, E, Del Grosso, V, Cappello, B, Genazzani, AA, Huttner, B, Leufkens, HGM et al.. Medicines not recommended for inclusion in the who essential medicines list: a retrospective observational study. Front Med (Lausanne). 2025;12 :1517020. doi: 10.3389/fmed.2025.1517020. PubMed PMID:40166059 PubMed Central PMC11955596.
  4. Yu, SR. Improving the Reimbursement Process for New Drugs: A Case Study of a Two-Waiver System in South Korea. J Eval Clin Pract. 2025;31 (3):e70074. doi: 10.1111/jep.70074. PubMed PMID:40165596 PubMed Central PMC11959314.
  5. Aksoyalp, ZS, Kayki-Mutlu, G, Wojnowski, L, Michel, MC. A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2024. Naunyn Schmiedebergs Arch Pharmacol. 2025; :. doi: 10.1007/s00210-025-04020-2. PubMed PMID:40163152 .
  6. van den Berg, S, Hollak, CE, Timmers, L, de Visser, SJ, De Visser, SJ. Twenty-Four Years after launch of the EU Orphan Regulation: Analyzing Dutch Price Dynamics, Biosimilars and Generics for Orphan Medicinal Products. Value Health. 2025; :. doi: 10.1016/j.jval.2025.03.004. PubMed PMID:40154839 .
  7. Wilson, K, Chiodi, F, Paine, A, Hakimi, Z, Ward, V, Macmillan, T et al.. Loncastuximab Tesirine Versus Polatuzumab Vedotin Plus Bendamustine and Rituximab in Relapsed/Refractory DLBCL After ≥ 2 Lines of Therapy: Matching-Adjusted Indirect Comparison. Adv Ther. 2025; :. doi: 10.1007/s12325-025-03169-9. PubMed PMID:40153222 .
  8. Occhipinti, G, Brugaletta, S, Abbate, A, Pedicino, D, Del Buono, MG, Vinci, R et al.. Inflammation in coronary atherosclerosis: diagnosis and treatment. Heart. 2025; :. doi: 10.1136/heartjnl-2024-325408. PubMed PMID:40139681 .
  9. Lexchin, J. Therapeutic value of oncology products with a conditional approval from Health Canada: a cross-sectional study. JRSM Open. 2025;16 (3):20542704251325314. doi: 10.1177/20542704251325314. PubMed PMID:40124210 PubMed Central PMC11924082.
  10. Tataru, EA, Dooms, M, Gonzaga-Jauregui, C, Pasmooij, AMG, O'Connor, DJ, Jonker, AH et al.. Drug-device combinations in rare diseases: Challenges and opportunities. Drug Discov Today. 2025;30 (4):104343. doi: 10.1016/j.drudis.2025.104343. PubMed PMID:40122448 .
  11. Deesker, LJ, Franssen, CFM, Dorresteijn, E, van de Kar, NCAJ, Nurmohamed, SA, Severs, D et al.. Controlled access to lumasiran in primary hyperoxaluria type 1: evaluation of a new access route for orphan drugs in The Netherlands. Nephrol Dial Transplant. 2025; :. doi: 10.1093/ndt/gfaf060. PubMed PMID:40121182 .
  12. Awad, IA, Alcazar-Felix, RJ, Stadnik, A, Kinkade, S, Jhaveri, A, Lee, J et al.. Safety and efficacy of atorvastatin for rebleeding in cerebral cavernous malformations (AT CASH EPOC): a phase 1/2a, randomised placebo-controlled trial. Lancet Neurol. 2025;24 (4):295-304. doi: 10.1016/S1474-4422(25)00036-5. PubMed PMID:40120614 .
  13. Szabó, R, Hornyánszky, Á, Kiss, DJ, Keserű, GM. Fluorescent tools for imaging class A G-protein coupled receptors. Eur J Pharm Sci. 2025;209 :107074. doi: 10.1016/j.ejps.2025.107074. PubMed PMID:40113106 .
  14. Breccia, M, Palandri, F, Martelli, M, Mendicino, F, Malato, A, Palumbo, GA et al.. Dosing and clinical outcomes of ruxolitinib in patients with myelofibrosis in a real-world setting: Interim results of the Italian observational study (ROMEI). Cancer. 2025;131 (7):e35801. doi: 10.1002/cncr.35801. PubMed PMID:40111826 PubMed Central PMC11925231.
  15. Scholte, SJ, Yaqub, O. Making modafinil: Classification and serendipity in drug development. Soc Stud Sci. 2025; :3063127251322113. doi: 10.1177/03063127251322113. PubMed PMID:40099909 .
  16. 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 PubMed Central PMC11906515.
  17. 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; :1-7. doi: 10.1080/14737167.2025.2479131. PubMed PMID:40077941 .
  18. 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.
  19. Jaksa, A, Arena, PJ, Hanisch, M, Marsico, M. Use of Real-World Evidence in Health Technology Reassessments Across 6 Health Technology Assessment Agencies. Value Health. 2025; :. doi: 10.1016/j.jval.2025.02.012. PubMed PMID:40054770 .
  20. 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 PubMed Central PMC11920007.
Search PubMed