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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.
Age
6 - No limit years
Sex
ALL
Healthy Volunteers
No
Carolinas Medical Center - Pediatrics
Charlotte, North Carolina, United States
Start Date
November 1, 2011
Primary Completion Date
July 1, 2017
Completion Date
July 1, 2017
Last Updated
April 28, 2022
13
ACTUAL participants
Lead Sponsor
Wake Forest University Health Sciences
Collaborators
Data Source & Attribution
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