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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: kalland me. 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 article.
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: kalland me. 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.
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: kalland me. Front Pharmacol. 2022 Aug 11;13:920336. doi: 10.3389/fphar.2022.920336. eCollection 2022. Front Pharmacol. 2022. PMID: 36034814 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. 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 Medicines Agency Review of Kymriah (Tisagenlecleucel) for the Treatment of Acute Lymphoblastic Leukemia and Diffuse Large B-Cell Lymphoma.
Ali S, Kjeken R, Niederlaender C, Markey G, Saunders TS, Opsata M, Moltu K, Bremnes B, Grønevik E, Muusse M, Håkonsen GD, Skibeli V, Kalland ME, Wang I, Buajordet I, Urbaniak A, Johnston J, Rantell K, Kerwash E, Schuessler-Lenz M, Salmonson T, Bergh J, Gisselbrecht C, Tzogani K, Papadouli I, Pignatti F. Ali S, et al. Among authors: kalland me. Oncologist. 2020 Feb;25(2):e321-e327. doi: 10.1634/theoncologist.2019-0233. Epub 2019 Oct 16. Oncologist. 2020. PMID: 32043764 Free PMC article. Review.
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