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Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease.
Vite CH, Bagel JH, Swain GP, Prociuk M, Sikora TU, Stein VM, O'Donnell P, Ruane T, Ward S, Crooks A, Li S, Mauldin E, Stellar S, De Meulder M, Kao ML, Ory DS, Davidson C, Vanier MT, Walkley SU. Vite CH, et al. Sci Transl Med. 2015 Feb 25;7(276):276ra26. doi: 10.1126/scitranslmed.3010101. Sci Transl Med. 2015. PMID: 25717099 Free PMC article.
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.
Gurda BL, De Guilhem De Lataillade A, Bell P, Zhu Y, Yu H, Wang P, Bagel J, Vite CH, Sikora T, Hinderer C, Calcedo R, Yox AD, Steet RA, Ruane T, O'Donnell P, Gao G, Wilson JM, Casal M, Ponder KP, Haskins ME. Gurda BL, et al. Among authors: bagel j. Mol Ther. 2016 Feb;24(2):206-216. doi: 10.1038/mt.2015.189. Epub 2015 Oct 8. Mol Ther. 2016. PMID: 26447927 Free PMC article.
Cerebrospinal Fluid Calbindin D Concentration as a Biomarker of Cerebellar Disease Progression in Niemann-Pick Type C1 Disease.
Bradbury A, Bagel J, Sampson M, Farhat N, Ding W, Swain G, Prociuk M, O'Donnell P, Drobatz K, Gurda B, Wassif C, Remaley A, Porter F, Vite C. Bradbury A, et al. Among authors: bagel j. J Pharmacol Exp Ther. 2016 Aug;358(2):254-61. doi: 10.1124/jpet.116.232975. Epub 2016 Jun 15. J Pharmacol Exp Ther. 2016. PMID: 27307499 Free PMC article.
137 results