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.
September 2025 Update
Applied Therapeutics has completed a meeting with the U.S. Food and Drug Administration to discuss a potential new drug application for govorestat in CMT-SORD. The company is awaiting official meeting minutes to determine next steps. CMTA continues to follow these developments. Read Applied Therapeutics’ press release here.
