Limb-girdle Muscular Dystrophy research hub
3 indexed studies · 1 currently recruiting
- active not recruiting
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
This study looks at how certain types of Limb-girdle Muscular Dystrophy (LGMD) progress over time in people aged 4 and older. It observes their mobility and lung function to understand natural changes in the condition, without testing any new treatments.
United States · Belgium · Brazil - active not recruitingPHASE3
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
This study is testing an experimental gene therapy, SRP-9003, for Limb-girdle Muscular Dystrophy type 2E/R4 (LGMD2E/R4). It aims to see how a single dose affects a specific gene in people who can walk and those who can't.
United States · Belgium · Germany - recruiting
LGMD R1 Natural History Study
This study is looking at how a specific type of muscular dystrophy, called Limb Girdle Muscular Dystrophy R1 (or LGMD2A), affects people over time. It's an observational study, meaning researchers will watch and learn without giving any new treatments.
United States · Netherlands · United Kingdom