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Clinical Trial Summary

This study will follow participants who are screened and confirmed with a genetic diagnosis of Limb-girdle muscular dystrophy type 2E (LGMD2E/R4), Limb-girdle muscular dystrophy type 2D (LGMD2D/R3), Limb-girdle muscular dystrophy type 2C (LGMD2C/R5), or Limb-girdle muscular dystrophy type 2A (LGMD2A/R1). These enrolled participants will be followed to evaluate mobility and pulmonary function for up to 3 years after enrollment. Additional participant data will be collected from the time the individual began experiencing LGMD symptoms to the present.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04475926
Study type Observational
Source Sarepta Therapeutics, Inc.
Contact Sarepta Therapeutics Inc., For Clinical Trial Information, Selec
Phone 1-888-SAREPTA (1-888-727-3782)
Email SareptAlly@sarepta.com
Status Recruiting
Phase
Start date April 22, 2021
Completion date March 31, 2028

See also
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