Assessment of Gross Motor Function in Alexander Disease (AxD) (P10-6.003)
To evaluate feasibility and performance of motor clinical outcome assessments (COAs) in the Alexander disease (AxD) for application in future natural history studies and clinical trials.
Authors
- Tracy Kornafel
- Walter Faig (ORCID: https://orcid.org/0000-0002-8676-3847)
- Joshua Joung (ORCID: https://orcid.org/0009-0000-4151-4655)
- Amy Tara Waldman (ORCID: https://orcid.org/0000-0002-1054-0815)
- Geraldine Liu
- Francesco Gavazzi (ORCID: https://orcid.org/0000-0002-4908-8395)
- Samuel R. Pierce (ORCID: https://orcid.org/0000-0003-3215-4162)
- Kathryn Gallison
Institutions
- Children's Hospital of Philadelphia (US)
- Neurology, Inc (US)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000211251
- Primary Topic
- RNA regulation and disease
- Type
- article
- Field-Weighted Citation Impact
- 0.00