Experimental gene therapy targets rare form of duchenne muscular dystrophy
Disease control
Completed
This early-stage trial tested a single-dose gene therapy called scAAV9.U7.ACCA in 3 boys with Duchenne muscular dystrophy caused by a duplication of exon 2. The therapy was given through a vein and aimed to help muscle cells produce dystrophin, a protein missing in this disease. …
Phase: PHASE1, PHASE2 • Sponsor: Megan Waldrop • Aim: Disease control
Last updated Jun 26, 2026 16:38 UTC