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AT-007 in Patients with Sorbitol Dehydrogenase (SORD) Deficiency
2024-518250-16-00Phase 3Completed
- Sponsor
- Applied Therapeutics, Inc.
- Start
- Oct 2022
- Modality
- Small Molecules
- Countries
- 4
01
Overview
Phase 3, randomised, double-blind trial in Charcot-Marie-Tooth disease type 2S (CMT2S), Charcot-Marie-Tooth disease type 2 (CMT2) and distal hereditary motor neuronopathy (dHMN) comparing AT-007 (GOVORESTAT) with Placebo (2 arms). Primary endpoints: Change in blood sorbitol at Month 12 and Change in 10MWRT at Month 24. Started Oct 2022; status: completed.
02
Drugs
govorestat
03
Conditions
Charcot-Marie-Tooth disease type 2S (CMT2S)Charcot-Marie-Tooth disease type 2 (CMT2)distal hereditary motor neuronopathy (dHMN)
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