Development of a General Composite Scale (GENCOMS) for Progressive Neurodegenerative Diseases and Implications for the Assessment of Disease-Modifying Therapies
The methodology presented here provides a generalizable framework for developing composite scales in the assessment of neurodegenerative disease progression and evaluation of DMT effects. By objectively selecting and weighting items from previously validated measures based solely on their sensitivity to disease progression, this methodology allows for the creation of a more responsive measurement of clinical decline. This heightened sensitivity to clinical decline can be utilized to detect modest yet meaningful treatment effects in the early stages of neurogenerative diseases, when it is optimal to begin a DMT.
Authors
- Suzanne Hendrix (ORCID: https://orcid.org/0000-0002-2374-7727)
- Craig Mallinckrodt
- Basia Rogula (ORCID: https://orcid.org/0000-0002-2115-8275)
- Michele Potashman (ORCID: https://orcid.org/0000-0001-7389-3354)
- Vladimir Coric
- Samuel P. Dickson (ORCID: https://orcid.org/0000-0002-4622-1349)
- Gilbert L’Italien
- Lauren C Powell
Institutions
- Vancouver Coastal Health (CA)
- Biohaven Pharmaceuticals (United States) (US)
- TetraLogic Pharmaceuticals (United States) (US)
Publication Details
- Journal
- Neurology and Therapy
- Published
- 2024-09-17
- DOI
- https://doi.org/10.1007/s40120-024-00661-2
- Citations
- 3
- Primary Topic
- Genetic Neurodegenerative Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.80