Overview
- The trial will assess safety and whether the investigational therapy can slow disease progression and improve hearing.
- Sites include Jichi Medical University Hospital in Tochigi Prefecture as part of a four-facility network.
- Researchers described mitochondrial disease as a rapidly progressive, severe rare condition with significant unmet need.
- Professor Takaaki Abe said successful drug development could offer hope to patients and families.
- The team noted mitochondrial dysfunction also features in age-related and noise-induced hearing loss, suggesting potential wider relevance if benefits are observed.