Limb Girdle Muscular Dystrophy (LGMD) Natural History
| Status: | Completed |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 6 - Any |
| Updated: | 7/20/2017 |
| Start Date: | November 2011 |
| End Date: | July 2017 |
Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy
The purpose of this study is to understand more about limb-girdle muscular dystrophy.
Therefore, the investigators would like to track the following information collected once a
year from patients with GENETICALLY CONFIRMED LGMD: quality of life questionnaires, muscle
strength, motor function, routine examination, assessment of patient (or parent)
understanding of LGMD, and serum (blood) for growth factors, cytokines, and biomarkers (all
parts of your blood). By tracking this information, we hope to be able to understand more
about the diagnosis, progression and natural history of this disorder.
Therefore, the investigators would like to track the following information collected once a
year from patients with GENETICALLY CONFIRMED LGMD: quality of life questionnaires, muscle
strength, motor function, routine examination, assessment of patient (or parent)
understanding of LGMD, and serum (blood) for growth factors, cytokines, and biomarkers (all
parts of your blood). By tracking this information, we hope to be able to understand more
about the diagnosis, progression and natural history of this disorder.
Inclusion Criteria:
- You have a GENETICALLY CONFIRMED diagnosis of Limb Girdle Muscular Dystrophy
- You must be at least 6 years of age or older (if under 18 you will need Parental
consent)
- You must be able to travel to the study site
- You must be able to provide a DNA/Gene testing report that confirms a diagnosis of
LGMD
Exclusion Criteria:
- You or your child do not have a diagnosis of LGMD
- Your child is under age 6
- You or your child are not able to travel to the study site
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