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A single-patient investigator-initiated trial of AT-007 in PMM2-CDG

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Trial Profile

A single-patient investigator-initiated trial of AT-007 in PMM2-CDG

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 26 May 2026

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At a glance

  • Drugs Govorestat (Primary)
  • Indications Congenital disorders of glycosylation
  • Focus Adverse reactions

Most Recent Events

  • 13 Nov 2025 According to an Applied therapeutics media release, results of Single-Patient Investigator-Initiated Trial Evaluating the Treatment of PMM2-CDG with Govorestat Published in Journal of Inherited Metabolic Disease (JIMD) and Presented at the 2025 American Society of Human Genetics (ASHG) Annual Meeting.
  • 12 Nov 2020 According to an Applied therapeutics media release, the company expects to initiate this study in H1 2021.
  • 28 Sep 2020 New trial record

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