Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA
Status:
Completed
Trial end date:
2019-03-14
Target enrollment:
Participant gender:
Summary
Dose escalation study of self-complementary (sc) AAVrh74.tMCK.hSGCA delivered to single leg
(low dose n=2) and bilaterally (n=6) via a major lower limb artery to the whole lower limb of
limb girdle muscular dystrophy type 2D (LGMD2D; alpha-sarcoglycan deficient) patients.