How to use the speech recognition tool?
Safety and efficacy of a single subretinal injection of rAAV.hCNGA3 in patients with CNGA3-linked achromatopsia investigated in an exploratory, dose-escalation trial.
Status of the trial: Active, Recruiting
Orphan Drug Recognition: Yes
- Opening Date: 2014-09-12
- Closing Date: 2027-04-01
Inclusion: clinical diagnosis of achromatopsia, age ≥ 18 years, confirmed mutation in CNGA3, BCVA ≥ 20/400; first cohort: BCVA ≤ 20/160, area of residual outer nuclear layer thickness in OCT scans of posterior pole, ability to understand and willingness to consent t
Funder Type: other
HCP: Principal investigators
HCP: Other investigators
Within ERN-EYE members
- Retinal Rare Eye Diseases (WG1)
This ERN is supported by: