Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency
Status:
Recruiting
Trial end date:
2026-07-31
Target enrollment:
Participant gender:
Summary
This study is designed to assess the efficacy and safety of AT-007 treatment in patients with
SORD Deficiency. This randomized, double-blind study will assess the effect of AT-007
compared to Placebo in SORD Deficiency patients for 24 months.