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Antisense drugs for rare and ultra-rare genetic neurological diseases.
- Source :
-
Neuron [Neuron] 2023 Aug 16; Vol. 111 (16), pp. 2465-2468. Date of Electronic Publication: 2023 Jun 23. - Publication Year :
- 2023
-
Abstract
- The regulatory approvals of nusinersen and tofersen, plus the large body of clinical and preclinical data from other drugs, have significantly de-risked antisense technology for neurological diseases. The platform learnings over the last 2 decades can be applied to subsequent drugs to improve the efficiency of discovering effective neuro-therapeutics.<br />Competing Interests: Declaration of interests Madelyn E. McCauley and C. Frank Bennett are employees and shareholders of Ionis Pharmaceuticals, Inc.<br /> (Copyright © 2023 Elsevier Inc. All rights reserved.)
Details
- Language :
- English
- ISSN :
- 1097-4199
- Volume :
- 111
- Issue :
- 16
- Database :
- MEDLINE
- Journal :
- Neuron
- Publication Type :
- Academic Journal
- Accession number :
- 37354903
- Full Text :
- https://doi.org/10.1016/j.neuron.2023.05.027