welcome to DuchenneXchange

- a positively charged Duchenne muscular dystrophy community.
  • join today!
active, not recruiting

Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

key information

study id #: NCT03333590

condition: Duchenne Muscular Dystrophy

status: active, not recruiting


The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients. There will be a modified intravascular limb infusion (ILI) procedure that will be used to sequentially deliver vector to each whole lower limb of DMD subjects via a major lower limb artery.

intervention: rAAVrh74.MCK.GALGT2

mechanism of action: Gene therapy to introduce a version of dystrophin

results: https://clinicaltrials.gov/ct2/show/results/NCT03333590

last updated: February 27, 2020