A Study of the Natural History of Participants With LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, and LGMD2A/R1 ≥ 4 Years of Age, Who Are Managed in Routine Clinical Practice

LGMD Type 2A, 2C, and 2D cohorts are now closed. Enrollment in the LGMD Type 2E cohort is closed and participants will be followed for up to five years.

Primary outcomes

• NSAD Total Score [Baseline up to Month 60]
• Time to Rise from the Floor [Baseline up to Month 60]
• Time of 10-Meter Walk/Run [10MWR] [Baseline up to Month 60]
• Time to Ascend 4 Steps [Baseline up to Month 60]
• Ankle Range of Motion (ROM) [Baseline up to Month 60]
• Dimension of the Performance of the Upper Limb (PUL) [Baseline up to Month 60]
• Timed Up and Go (TUG) [Baseline up to Month 60]
• Time of 100-Meter Walk/Run (100MWR) [Baseline up to Month 60]
• Pulmonary Function Test: Forced Vital Capacity (FVC) [Baseline up to Month 60]

Sex: All, ages: 4 Years to any

Inclusion Criteria:

* Male or female participant ≥ 4 years of age who demonstrate symptoms of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1 in the opinion of the investigator (eg, muscle weakness, loss of function, delayed milestones).
* Confirmed clinical and genetic diagnosis of LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, or LGMD2A/R1.

Exclusion Criteria:

* Demonstrates cognitive delay or impairment that could confound motor development, in the opinion of the Investigator.
* Has a medical condition, in the opinion of the Investigator, that might compromise participants ability to comply with study requirements.
* Is participating in other interventional study(ies) at the time of enrollment in this study.

• Medical Director, Study director, Sarepta Therapeutics, Inc.