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Nusinersen: A Review in 5q Spinal Muscular Atrophy
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“Nusinersen is designed to modify the splicing of the SMN2 (Survival of motor neuron 2, centromeric) gene, increasing the production of functional SMN protein.184,186,187”
From MiR-128-3p – a gray eminence of the human central nervous system (2024) · cites this paper as [186]
In the citing paper
“The U.S. Food and Drug Administration approved the first CNS-related oligonucleotide-based drug, Nusinersen, for treating spinal muscular atrophy caused by a survival motor neuron (SMN) protein deficiency.”
From MiR-128-3p – a gray eminence of the human central nervous system (2024) · cites this paper as [186]
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Citations
Cites
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
Finkel, R. S., Mercuri, E., Darras, B. T., Connolly, A. M., Kuntz, N. L., Kirschner, J., Chiriboga, C. A., Saito, K., Servais, L., Tizzano, E., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1702752 · Cited by 2,337
Onasemnogene Abeparvovec: First Global Approval
Hoy, S. M. (2019) · Drugs · doi:10.1007/s40265-019-01162-5 · Cited by 337
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Research works printed but not linked: 53
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Cited by
Sorted by citations
MiR-128-3p – a gray eminence of the human central nervous system
Kiel, K., Król, S. K., Bronisz, A., & Godlewski, J. (2024) · Molecular Therapy Nucleic Acids · doi:10.1016/j.omtn.2024.102141 · Cited by 15
Metadata
- Authors
- Hoy, S. M.
- Venue
- CNS Drugs
- Published
- 2021-11-30
- Volume
- 35
- Issue
- 12
- Pages
- 1317-1328
- ISSN
- 1172-7047
- 1179-1934
- PMC
- PMC8709816
- Cited by
- 50
- References
- 2
- Language
- en
- Update notices
- correction (2021-12-27)
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