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Nonclinical data supporting orphan medicinal product designations: lessons from rare neurological conditions.
Sheean ME, Stoyanova-Beninska V, Capovilla G, Duarte D, Hofer MP, Hoffmann M, Magrelli A, Mariz S, Tsigkos S, Shaili E, Polsinelli B, Ricciardi M, Bonelli M, Balabanov P, Larsson K, Sepodes B. Sheean ME, et al. Drug Discov Today. 2018 Jan;23(1):26-48. doi: 10.1016/j.drudis.2017.09.015. Epub 2017 Oct 4. Drug Discov Today. 2018. PMID: 28987289 Review.
The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products.
Palomo GM, Pose-Boirazian T, Naumann-Winter F, Costa E, Duarte DM, Kalland ME, Malikova E, Matusevicius D, Vitezic D, Larsson K, Magrelli A, Stoyanova-Beninska V, Mariz S. Palomo GM, et al. Among authors: duarte dm. Mol Ther. 2023 Dec 6;31(12):3414-3423. doi: 10.1016/j.ymthe.2023.09.020. Epub 2023 Oct 4. Mol Ther. 2023. PMID: 37794679 Free PMC article.
Advancing rare disease treatment: EMA's decade-long insights into engineered adoptive cell therapy for rare cancers and orphan designation.
Kalland ME, Pose-Boirazian T, Palomo GM, Naumann-Winter F, Costa E, Matusevicius D, Duarte DM, Malikova E, Vitezic D, Larsson K, Magrelli A, Stoyanova-Beninska V, Mariz S. Kalland ME, et al. Among authors: duarte dm. Gene Ther. 2024 Jul;31(7-8):366-377. doi: 10.1038/s41434-024-00446-0. Epub 2024 Mar 14. Gene Ther. 2024. PMID: 38480914 Free PMC article. Review.
Nonclinical data supporting orphan medicinal product designations in the area of rare infectious diseases.
Sheean ME, Malikova E, Duarte D, Capovilla G, Fregonese L, Hofer MP, Magrelli A, Mariz S, Mendez-Hermida F, Nistico R, Leest T, Sipsas NV, Tsigkos S, Vitezic D, Larsson K, Sepodes B, Stoyanova-Beninska V. Sheean ME, et al. Drug Discov Today. 2020 Feb;25(2):274-291. doi: 10.1016/j.drudis.2019.10.015. Epub 2019 Nov 5. Drug Discov Today. 2020. PMID: 31704277 Free article. Review.
32 results