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 (EMERGENE)

Open to people ages 4 years and up

• Cohort 1, only ambulatory participants:
  • Able to walk without assistive aid
  • 10-meter walk test (10MWT) <30 seconds
  • NSAD ≥25
• Cohort 2, only non-ambulatory participants:
  • 10MWT ≥30 seconds or unable to perform
  • PUL 2.0 entry scale score ≥3
• Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
• Able to cooperate with muscle testing
• Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.

• Left ventricular ejection fraction < 40% or clinical signs and/or symptoms of cardiomyopathy
• Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
• Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
• Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)

Other inclusion/exclusion criteria apply.