Long-Term Study of Muscle Disease Outcome Measures
Follows people with limb girdle, myotonic dystrophy type 2 or late-onset Pompe disease.
Plain-language title generated from the registry record
Registry title: Long-Term Development of Muscular Dystrophy Outcome Assessments
Key facts
- Where
- Richmond, United StatesSee the site
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 6 and 50 years old?
Source: NCT05989620, who can take part
Where in United States
- Virginia Commonwealth UniversityRichmond, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Virginia Commonwealth University
- Trial code
- NCT05989620
- Record updated
- 18 November 2025
- Original description
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).Read full original descriptionShow less
Long-Term Development of Muscular Dystrophy Outcome Assessments (GRASP-01-005)
This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).
Inclusion: Age between 6-50 years at enrollment; Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with proximal weakness); Genetic confirmation of a LGMD, DM2, or LOPD; FVC above 30% of predicted.
Exclusion: Participation in a clinical trial receiving an investigational product; Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator.