Limb Girdle Muscular Dystrophy (LGMD) Natural History

Wake Forest University Health Sciences13 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)

Eligibility

Sex: ALLMin age: 6 Years

Medical Language ↔ Plain English

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

Limb Girdle Muscular Dystrophy (LGMD) Natural History

Overview

Conditions

Eligibility

Locations (1)

Outcomes

Primary Outcomes