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
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Sarepta Therapeutics, Inc.
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.
Age
4–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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.
Clareo Health | 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