The dose escalation trial evaluated the effects of a single intrathecal injection of four escalating doses of scAAV9/JeT-GAN in 14 patients over the age of six with GAN-related giant axon neuropathy.
- There were very few serious side-effects attributable to the treatment during the trial’s average follow-up of five and a half years.
- One of the doses tested resulted in a significant slowing of muscle damage as measured by MFM-32.
- Partial restoration of sensory response in the upper limbs was observed in six participants six to 24 months after treatment.
