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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. Among authors: magrelli a. 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.
Defining Satisfactory Methods of Treatment in Rare Diseases When Evaluating Significant Benefit-The EU Regulator's Perspective.
Sheean ME, Naumann-Winter F, Capovilla G, Kalland ME, Malikova E, Mariz S, Matusevicius D, Nistico R, Schwarzer-Daum B, Tsigkos S, Tzogani K, Larsson K, Magrelli A, Stoyanova-Beninska V. Sheean ME, et al. Among authors: magrelli a. Front Med (Lausanne). 2021 Aug 27;8:744625. doi: 10.3389/fmed.2021.744625. eCollection 2021. Front Med (Lausanne). 2021. PMID: 34513895 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: magrelli a. 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.
The role of microRNAs in the biology of rare diseases.
Salvatore M, Magrelli A, Taruscio D. Salvatore M, et al. Among authors: magrelli a. Int J Mol Sci. 2011;12(10):6733-42. doi: 10.3390/ijms12106733. Epub 2011 Oct 11. Int J Mol Sci. 2011. PMID: 22072915 Free PMC article. Review.
38 results