Long-Term Development of Muscular Dystrophy Outcome Assessments

Summary

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).

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

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

Conditions30

Locations1 site

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