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Providing high-quality care remotely to patients with rare bone diseases during COVID-19 pandemic.
Brizola E, Adami G, Baroncelli GI, Bedeschi MF, Berardi P, Boero S, Brandi ML, Casareto L, Castagnola E, Fraschini P, Gatti D, Giannini S, Gonfiantini MV, Landoni V, Magrelli A, Mantovani G, Michelis MB, Nasto LA, Panzeri L, Pianigiani E, Scopinaro A, Trespidi L, Vianello A, Zampino G, Sangiorgi L. Brizola E, et al. Among authors: magrelli a. Orphanet J Rare Dis. 2020 Aug 31;15(1):228. doi: 10.1186/s13023-020-01513-6. Orphanet J Rare Dis. 2020. PMID: 32867855 Free PMC article.
Navigating the orphan medicinal product designation: Evidence requirements for gene therapies in Europe.
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: magrelli a. Mol Ther. 2024 Oct 28:S1525-0016(24)00675-0. doi: 10.1016/j.ymthe.2024.10.015. Online ahead of print. Mol Ther. 2024. PMID: 39489919 Free 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 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: magrelli a. 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.
Licensing of Orphan Medicinal Products-Use of Real-World Data and Other External Data on Efficacy Aspects in Marketing Authorization Applications Concluded at the European Medicines Agency Between 2019 and 2021.
Naumann-Winter F, Wolter F, Hermes U, Malikova E, Lilienthal N, Meier T, Kalland ME, Magrelli A. Naumann-Winter F, et al. Among authors: magrelli a. Front Pharmacol. 2022 Aug 11;13:920336. doi: 10.3389/fphar.2022.920336. eCollection 2022. Front Pharmacol. 2022. PMID: 36034814 Free PMC article.
38 results