Gene therapy injection aims to restore swallowing in rare muscle disorder
Disease control
Recruiting now
This study tests a single injection of BB-301 into throat muscles to improve swallowing in people with oculopharyngeal muscular dystrophy (OPMD). About 30 adults aged 50-65 who have already been in a natural history study will receive the treatment during a short surgery. The mai…
Phase: PHASE1, PHASE2 • Sponsor: Benitec Biopharma, Inc. • Aim: Disease control
Last updated Apr 30, 2026 15:48 UTC