Long-Term Development of Muscular Dystrophy Outcome Assessments
Recruiting
Observational Study
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
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 6 – 50
- Sex
- Any
- Study type
- Observational
- Participants needed
- 1,000 (estimated)
- Sponsor
- Virginia Commonwealth University · Other
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About This Trial
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).
Trial Locations
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Eligibility Criteria
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…
Contacts