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RecruitingLGMD1BLGMD1CLGMD1D

Long-Term Development of Muscular Dystrophy Outcome Assessments

Eligible age

6–50 yrs

Accepts

All genders

Locations

1 state

Healthy volunteers

No

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About this study

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).

Sponsor: Virginia Commonwealth University

You may qualify if…

  • 1. Age between 6-50 years at enrollment
  • 2. Clinically affected (defined as weakness on bedside evaluation in a pattern consistent with proximal weakness)
  • 3. Genetic confirmation of a LGMD, DM2, or LOPD
  • 4. FVC above 30% of predicted

You may not qualify if…

  • 1. 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
  • 2. Participation in a clinical trial receiving an investigational product

Where it's recruiting

Virginia

Richmond

Source: ClinicalTrials.gov · NCT05989620 · last updated 2025-11-18

Long-Term Development of Muscular Dystrophy Outcome Assessments · TrialPath