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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
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Cites
Age-related Volumetric Changes of Brain Gray and White Matter in Healthy Infants and Children
Matsuzawa, J. (2001) · Cerebral Cortex · doi:10.1093/cercor/11.4.335 · Cited by 315
Rigo, F., Chun, S. J., Norris, D. A., Hung, G., Lee, S., Matson, J., Fey, R. A., Gaus, H., Hua, Y., Grundy, J. S., et al. (2014) · The Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.113.212407 · Cited by 283
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Kulkarni, J. A., Witzigmann, D., Thomson, S. B., Chen, S., Leavitt, B. R., Cullis, P. R., & van der Meel, R. (2021) · Nature Nanotechnology · doi:10.1038/s41565-021-00898-0 · Cited by 1,371
Tau-targeting therapies for Alzheimer disease
Congdon, E. E., & Sigurdsson, E. M. (2018) · Nature Reviews Neurology · doi:10.1038/s41582-018-0013-z · Cited by 1,113
Crooke, S. T., Witztum, J. L., Bennett, C. F., & Baker, B. F. (2018) · Cell Metabolism · doi:10.1016/j.cmet.2018.03.004 · Cited by 841
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Rinaldi, C., & Wood, M. J. A. (2018) · Nature Reviews Neurology · doi:10.1038/nrneurol.2017.148 · Cited by 788
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Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice
Becker, L. A., Huang, B., Bieri, G., Ma, R., Knowles, D. A., Jafar-Nejad, P., Messing, J., Kim, H. J., Soriano, A., Auburger, G., et al. (2017) · Nature · doi:10.1038/nature22038 · Cited by 571
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Ringstad, G., Valnes, L. M., Dale, A. M., Pripp, A. H., Vatnehol, S.-A. S., Emblem, K. E., Mardal, K.-A., & Eide, P. K. (2018) · JCI Insight · doi:10.1172/jci.insight.121537 · Cited by 533
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Bennett, C. F., Krainer, A. R., & Cleveland, D. W. (2019) · Annual Review of Neuroscience · doi:10.1146/annurev-neuro-070918-050501 · Cited by 353
Self-citationThe glymphatic system: implications for drugs for central nervous system diseases
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Walgrave, H., Balusu, S., Snoeck, S., Vanden Eynden, E., Craessaerts, K., Thrupp, N., Wolfs, L., Horré, K., Fourne, Y., Ronisz, A., et al. (2021) · Cell Stem Cell · doi:10.1016/j.stem.2021.05.001 · Cited by 175
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Chen, X., Sanchis-Juan, A., French, C. E., Connell, A. J., Delon, I., Kingsbury, Z., Chawla, A., Halpern, A. L., Taft, R. J., Bentley, D. R., et al. (2020) · Genetics in Medicine · doi:10.1038/s41436-020-0754-0 · Cited by 137
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Crooke, S. T., Baker, B. F., Witztum, J. L., Kwoh, T. J., Pham, N. C., Salgado, N., McEvoy, B. W., Cheng, W., Hughes, S. G., Bhanot, S., et al. (2017) · Nucleic Acid Therapeutics · doi:10.1089/nat.2016.0650 · Cited by 129
Jafar-nejad, P., Powers, B., Soriano, A., Zhao, H., Norris, D. A., Matson, J., DeBrosse-Serra, B., Watson, J., Narayanan, P., Chun, S. J., et al. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkaa1235 · Cited by 128
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Chioccioli, M., Roy, S., Newell, R., Pestano, L., Dickinson, B., Rigby, K., Herazo-Maya, J., Jenkins, G., Ian, S., Saini, G., et al. (2022) · eBioMedicine · doi:10.1016/j.ebiom.2022.104304 · Cited by 129
(2018) · Handbook of Clinical Neurology · doi:10.1016/b978-0-444-63233-3.00017-8 · Cited by 122
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Mazur, C., Powers, B., Zasadny, K., Sullivan, J. M., Dimant, H., Kamme, F., Hesterman, J., Matson, J., Oestergaard, M., Seaman, M., et al. (2019) · JCI Insight · doi:10.1172/jci.insight.129240 · Cited by 114
Li, M., Jancovski, N., Jafar-Nejad, P., Burbano, L. E., Rollo, B., Richards, K., Drew, L., Sedo, A., Heighway, J., Pachernegg, S., et al. (2021) · Journal of Clinical Investigation · doi:10.1172/jci152079 · Cited by 115
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Bennett, C. F., Kordasiewicz, H. B., & Cleveland, D. W. (2021) · Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-010919-023738 · Cited by 108
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Annoussamy, M., Lilien, C., Gidaro, T., Gargaun, E., Chê, V., Schara, U., Gangfuß, A., D'Amico, A., Dowling, J. J., Darras, B. T., et al. (2019) · Neurology · doi:10.1212/wnl.0000000000007319 · Cited by 94
The ApoE receptors Vldlr and Apoer2 in central nervous system function and disease
Lane-Donovan, C., & Herz, J. (2017) · Journal of Lipid Research · doi:10.1194/jlr.r075507 · Cited by 94
Mapping genetic effects on cellular phenotypes with “cell villages”
Mitchell, J. M., Nemesh, J., Ghosh, S., Handsaker, R. E., Mello, C. J., Meyer, D., Raghunathan, K., de Rivera, H., Tegtmeyer, M., Hawes, D., et al. (2020) · doi:10.1101/2020.06.29.174383 · Cited by 63
Malong, L., Napoli, I., Casal, G., White, I. J., Stierli, S., Vaughan, A., Cattin, A.-L., Burden, J. J., Hng, K. I., Bossio, A., et al. (2023) · Developmental Cell · doi:10.1016/j.devcel.2023.01.002 · Cited by 59
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Mortberg, M. A., Gentile, J. E., Nadaf, N. M., Vanderburg, C., Simmons, S., Dubinsky, D., Slamin, A., Maldonado, S., Petersen, C. L., Jones, N., et al. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad371 · Cited by 54
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Hoy, S. M. (2021) · CNS Drugs · doi:10.1007/s40263-021-00878-x · Cited by 50
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Sasaki, S., & Guo, S. (2018) · Nucleic Acid Therapeutics · doi:10.1089/nat.2017.0696 · Cited by 24
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Tejwani, L., Jung, Y., Kokubu, H., Sowmithra, S., Ni, L., Lee, C., Sanders, B., Lee, P. J., Xiang, Y., Luttik, K., et al. (2023) · Journal of Clinical Investigation · doi:10.1172/jci138207 · Cited by 17
“spinal muscular atrophy (36) and Huntington’s disease (37).”
BackgroundSelf-citationProtein kinase inhibitors as therapeutics in neurodegenerative and psychiatric disorders
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Metadata
- Authors
- Finkel, R. S., Chiriboga, C. A., Vajsar, J., Day, J. W., Montes, J., De Vivo, D. C., Yamashita, M., Rigo, F., Hung, G., Schneider, E., Norris, D. A., Xia, S., Bennett, C. F., & Bishop, K. M.
- Venue
- The Lancet
- Publisher
- Elsevier BV
- Published
- 2016-12
- Volume
- 388
- Issue
- 10063
- Pages
- 3017-3026
- ISSN
- 0099-5355 (ISSN-L)
- 0140-6736
- OpenAlex
- W2560202024
- Cited by
- 1,019
- References
- 2
- FWCI
- 74.85
- Type
- journal article
- Language
- en
- Open access
- closed
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