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A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Long-Term Clinical Benefit of Govorestat in Patients With Charcot-Marie-Tooth-Sorbitol Dehydrogenase Deficiency (CMT-SORD)

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Trial Profile

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Long-Term Clinical Benefit of Govorestat in Patients With Charcot-Marie-Tooth-Sorbitol Dehydrogenase Deficiency (CMT-SORD)

Status: Not yet recruiting
Phase of Trial: Phase III

Latest Information Update: 30 Sep 2025

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At a glance

  • Drugs Govorestat (Primary)
  • Indications Charcot-Marie-Tooth disease; Charcot-Marie-Tooth disease type 2C
  • Focus Therapeutic Use
  • Sponsors Applied Therapeutics

Most Recent Events

  • 30 Sep 2025 New trial record
  • 13 Aug 2025 According to an Applied Therapeutics media release, the company is going to meet FDA to align on its planned NDA submission strategy for govorestat, Q3 2025. The company will discuss the latest comprehensive data generated on govorestat for the treatment of Sorbitol Dehydrogenase Deficiency (SORD), a subtype of Charcot-Marie-Tooth (CMT) disease.

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