Quantitative relationship between tominersen concentrations in cerebrospinal fluid and biomarker changes in Huntington's disease patients
The PKPD model quantitatively confirms the relationship between tominersen exposure and CSF mHTT lowering. The ER analysis suggests that lower tominersen exposure levels may offer a better benefit-risk profile.
Authors
- David J. Hawellek (ORCID: https://orcid.org/0000-0002-2529-3351)
- Marcus Björnsson
- Edward J. Wild (ORCID: https://orcid.org/0000-0002-6921-7887)
- Patricia Sanwald Ducray (ORCID: https://orcid.org/0000-0002-2904-5857)
- Sarah J. Tabrizi (ORCID: https://orcid.org/0000-0003-2716-2045)
- Peter McColgan (ORCID: https://orcid.org/0000-0002-5452-0648)
- Blair R. Leavitt (ORCID: https://orcid.org/0000-0002-4532-766X)
- Hanna E. Silber Baumann (ORCID: https://orcid.org/0000-0003-4928-6284)
- Paul Grimsey
- G. Bernhard Landwehrmeyer (ORCID: https://orcid.org/0000-0003-3375-790X)
- Karen Elta Anderson (ORCID: https://orcid.org/0000-0002-2084-6869)
- Yumi Yamamoto (ORCID: https://orcid.org/0000-0003-1798-3472)
Institutions
- Roche (Switzerland) (CH)
- University of British Columbia (CA)
- Georgetown University (US)
- Universität Ulm (DE)
- Roche (United Kingdom) (GB)
- National Hospital for Neurology and Neurosurgery (GB)
- University College London (GB)
Publication Details
- Journal
- British Journal of Clinical Pharmacology
- Published
- 2025-11-25
- DOI
- https://doi.org/10.1002/bcp.70367
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- CHDI Foundation
- F. Hoffmann-La Roche
- Ionis Pharmaceuticals
- Medical Research Council