Experimental gene therapy shows promise for rare form of duchenne muscular dystrophy
Disease control
Completed
This early-stage trial tested a gene therapy called scAAV9.U7.ACCA in 3 boys with Duchenne muscular dystrophy caused by a duplication of exon 2. The therapy was given as a single injection into a vein. The main goals were to check for serious side effects and to see if the treatm…
Phase: PHASE1, PHASE2 • Sponsor: Megan Waldrop • Aim: Disease control
Last updated May 17, 2026 12:54 UTC