Related Experiment Video
Updated: Oct 3, 2025
09:18
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
10.5K
Brief assessment of cognitive function in myotonic dystrophy: Multicenter longitudinal study using computer-assisted
Gayle K Deutsch1, Katharine A Hagerman1, Jacinda Sampson1
1Department of Neurology and Neurological Sciences, Stanford University School of Medicine, Stanford, CA.
Muscle & Nerve
|February 18, 2022
Summary
Computerized cognitive tests reliably assess central nervous system involvement in Myotonic Dystrophy type 1 (DM1) multicenter studies. Working memory deficits were prominent, suggesting utility for clinical trials.
Area of Science:
- Neuroscience
- Cognitive Science
- Clinical Research
Background:
- Myotonic Dystrophy type 1 (DM1) is known to impact cognitive function.
- Standardized methods for assessing central nervous system (CNS) involvement in multicenter DM1 studies are lacking.
Purpose of the Study:
- To evaluate the feasibility and reliability of computerized cognitive tests for assessing cognition in DM1.
- To identify cognitive domains most affected in DM1 for potential use in clinical trials.
Main Methods:
- A prospective, longitudinal, observational study involving 113 adults with DM1 across six sites.
- Assessment of psychomotor speed, attention, working memory, and executive functioning using computerized tests at baseline, 3, and 12 months.
- Comparison of cognitive data with muscle function and patient-reported outcomes (PROs).
Main Results:
- Computerized cognitive tests demonstrated moderate to good reliability for various cognitive domains.
- Working memory accuracy showed the poorest performance at baseline.
- Executive functioning improved from baseline to 3 months, potentially due to learning effects.
- A moderate correlation was observed between executive function and CTG repeat size.
Conclusions:
- Computerized cognitive tests are feasible and reliable for multicenter DM1 studies.
- Working memory deficits are a key finding, potentially serving as a valuable endpoint in clinical trials.
- Further research is needed to clarify the relationship between PROs and cognitive impairment in DM1.
