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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: mariz s. 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.
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: mariz s. 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: mariz s. 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 EMA Assessment of Asciminib for the Treatment of Adult Patients With Philadelphia Chromosome-Positive Chronic Myeloid Leukemia in Chronic Phase Who Were Previously Treated With at Least Two Tyrosine Kinase Inhibitors.
Tesileanu CMS, Michaleas S, Gonzalo Ruiz R, Mariz S, Fabriek BO, van Hennik PB, Dedorath J, Dekic B, Unkrig C, Brandt A, Koenig J, Enzmann H, Delgado J, Pignatti F. Tesileanu CMS, et al. Among authors: mariz s. Oncologist. 2023 Jul 5;28(7):628-632. doi: 10.1093/oncolo/oyad119. Oncologist. 2023. PMID: 37141403 Free PMC article. Clinical Trial.
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: mariz s. 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.
Regulatory Standards in Orphan Medicinal Product Designation in the EU.
Tsigkos S, Mariz S, Sheean ME, Larsson K, Magrelli A, Stoyanova-Beninska V. Tsigkos S, et al. Among authors: mariz s. Front Med (Lausanne). 2021 Jun 25;8:698534. doi: 10.3389/fmed.2021.698534. eCollection 2021. Front Med (Lausanne). 2021. PMID: 34249982 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. Among authors: mariz s. 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 orphan conditions in the context of the European orphan regulation: challenges and evolution.
O'Connor DJ, Sheean ME, Hofer MP, Tsigkos S, Mariz S, Fregonese L, Larsson K, Hivert V, Westermark K, Naumann-Winter F, Stoyanova-Beninska V, Barišić I, Capovilla G, Magrelli A, Sepodes B. O'Connor DJ, et al. Among authors: mariz s. Nat Rev Drug Discov. 2019 Jul;18(7):479-480. doi: 10.1038/nrd.2018.128. Nat Rev Drug Discov. 2019. PMID: 30940922 No abstract available.
50 results