One-Time gene infusion aims to fight duchenne muscular dystrophy
Disease control
Recruiting now
This study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to se…
Phase: PHASE1, PHASE2 • Sponsor: AO GENERIUM • Aim: Disease control
Last updated Jul 01, 2026 00:00 UTC