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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: palomo gm. 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. 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.
Parkin is a disease modifier in the mutant SOD1 mouse model of ALS.
Palomo GM, Granatiero V, Kawamata H, Konrad C, Kim M, Arreguin AJ, Zhao D, Milner TA, Manfredi G. Palomo GM, et al. EMBO Mol Med. 2018 Oct;10(10):e8888. doi: 10.15252/emmm.201808888. EMBO Mol Med. 2018. PMID: 30126943 Free PMC article.