AT-007, AT-007 Placebo, oral suspension clinical trials

A RandomIzed, Double-Blind, Placebo-CoNtrolled, Two-Part Study to Evaluate the Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients with SoRbitol Dehydrogenase (SORD) DEficiency

This study looks into a new medicine called AT-007 for people with SORD Deficiency. It compares AT-007 to a dummy medicine to see if it lowers sorbitol levels in the blood and improves how people walk and function over 1 to 2 years.