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Long-Term Development of Muscular Dystrophy Outcome Assessments

RECRUITING
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6 years to 50 years old
Inclusion Criteria:

• 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 Criteria:

• 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
• Participation in a clinical trial receiving an investigational product

LGMD1B, LGMD1C, LGMD1D, LGMD1E, LGMD1F, LGMD1G, LGMD1H, LGMD2A, LGMD2B, LGMD2C, LGMD2D, LGMD2E, LGMD2F, LGMD2G, LGMD2I, LGMD2J, LGMD2K, LGMD2L, LGMD2M, LGMD2N, LGMD2O, LGMD2P, LGMD2Q, LGMD2S, LGMD2T, LGMD2U, LGMD2W, LGMD2X, LGMD2Y

Muscular Dystrophy, Myotonic Dystrophy, Limb Girdle Muscular Dystrophy, LGMD, LOPD, Late Onset Pompe Disease

Jennifer Raymond - Jennifer.Raymond@vcuhealth.org
NCT05989620

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