A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects with Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)

Limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4) or β-sarcoglycanopathy

Change from Baseline in β-SG expression at Day 60 post-dose as measured by IF PβSGPF for subjects in Cohort 1 (ambulatory subjects)

Change from Baseline in β-SG expression at Day 60 post-dose as measured by IF PβSGPF for subjects in Cohort 1 (ambulatory subjects), Change from Baseline in β-SG expression at Day 60 post-dose for subjects in Cohorts 1 (ambulatory subjects) and 2 (non-ambulatory subjects) as measured by IF PFI, Change from Baseline in β-SG expression at Day 60 post-dose as measured by Western, Change from Baseline in β-SG expression at Day 60 post-dose as measured by IF PβSGPF for subjects in Cohort 2 (non-ambulatory subjects), Change from Baseline through Month 60 in the following physical function for subjects in Cohorts 1(ambulatory subject) and 2 (non-ambulatory subject) − NSAD total score − PUL 2.0 total score, Change from Baseline through Month 60 in the following timed function tests for subjects in Cohort 1 (ambulatory subject): − Time to rise from the floor − Time to complete the 10MWR − Time to ascend 4 steps − Time to complete the 100MWR − Timed up and go, Incidence of TEAEs, Incidence of adverse events of special interest, Incidence of treatment-emergent SAEs, Incidence of clinically significant laboratory abnormalities, Clinically significant changes in ECGs, ECHOs, Change from Baseline through Month 60 in CK level, Time to change of disease milestones

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