Test-Retest Reliability of Motor Function and Myometry Outcomes From the Vamorolone Trials in Duchenne Muscular Dystrophy
Reliable outcome measures, appropriate for the age of patients, are key to improving power in clinical trials, leading to fewer participants needed and reducing patient and site burden. We found NSAA, RWV, and CLIMBV to be the most reliable. This should be considered in the context of sensitivity to drug effect, and clinical meaningfulness of changes observed: all 5 motor outcomes were sensitive to drug effect of vamorolone and prednisone, whereas myometry was not.
Authors
- Rebecca Tobin (ORCID: https://orcid.org/0000-0001-7918-3193)
- Utkarsh J. Dang (ORCID: https://orcid.org/0000-0003-4120-2015)
- M. James (ORCID: https://orcid.org/0000-0001-8078-7744)
- Linda B. Johnson (ORCID: https://orcid.org/0009-0000-6901-8362)
- Eric P. Hoffman (ORCID: https://orcid.org/0000-0001-6470-5139)
- Paula R. Clemens (ORCID: https://orcid.org/0000-0002-0652-6703)
Institutions
- Binghamton University (US)
- Western Pennsylvania School for Blind Children (US)
- Muscular Dystrophy UK (GB)
- VA Pittsburgh Healthcare System (US)
- University of Pittsburgh Medical Center (US)
- Carleton University (CA)
Publication Details
- Journal
- Neurology Genetics
- Published
- 2025-08-29
- DOI
- https://doi.org/10.1212/nxg.0000000000200289
- Citations
- 1
- Primary Topic
- Muscle Physiology and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.81