Objective:
To evaluate the safety and efficacy of ATSN-201, an investigational gene therapy for X-linked retinoschisis (XLRS), in a clinical trial.
Key Findings:
- Structural and functional improvements were observed in treated adults.
- Foveal schisis closure was confirmed in four of six treated adults in Cohort 4.
- No serious adverse events were reported across all cohorts.
Interpretation:
Limitations:
- The study included a small sample size of nine adults and three pediatric patients.
- Control patients may receive treatment after one year of observation, which could affect outcomes.
Conclusion:
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