Targeting Huntingtin Expression in Patients with Huntington’s Disease
UK Dementia Research Institute · University College London
Abstract
messenger RNA and thereby reduce concentrations of mutant huntingtin.
pharmacokinetics in cerebrospinal fluid (CSF). Prespecified exploratory end points included the concentration of mutant huntingtin in CSF.
10-mg, 30-mg, 60-mg, 90-mg, and 120-mg dose groups, respectively).
to patients with early Huntington's disease was not accompanied by serious adverse events. We observed dose-dependent reductions in concentrations of mutant huntingtin. (Funded by Ionis Pharmaceuticals and F. Hoffmann-La Roche; ClinicalTrials.gov number, NCT02519036.).
Citation impact
- FWCI
- 45.09
- Percentile
- 100%
- References
- 26
Authors
22- SJSarah J. TabriziCorresponding
UK Dementia Research Institute, University College London
- BRBlair R. Leavitt
UK Dementia Research Institute, University College London
- GBG. Bernhard Landwehrmeyer
UK Dementia Research Institute, University College London
- EJEdward J. Wild
UK Dementia Research Institute, University College London
- CSCarsten Saft
UK Dementia Research Institute, University College London
Topics & keywords
- Huntingtin
- Disease
- Adverse effect
- Mutant
- Mutation
- Lysosomal storage disease