Limb Girdle Muscular Dystrophy (LGMD) Natural History

Wake Forest University (WFU)

Status: Completed

Conditions: Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)

Study type: Observational

Funder types: Other

Identifiers

NCT01783509

LGMD Nat Hx

Details and patient eligibility

About

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.

Enrollment: 13 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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

Trial design

13 participants in 1 patient group

LGMD

Description:

Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy