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A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Trial Profile

A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

Status: Not yet recruiting
Phase of Trial: Phase I

Latest Information Update: 10 Mar 2026

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At a glance

  • Drugs DNL 952 (Primary)
  • Indications Glycogen storage disease type II
  • Focus Adverse reactions
  • Sponsors Denali Therapeutics Inc

Most Recent Events

  • 06 Feb 2026 According to a Denali Therapeutics Inc media release, company announced that study design were presented presented this week at the 22nd Annual WORLDSymposium™ in San Diego, California.
  • 06 Feb 2026 According to a Denali Therapeutics Inc media release, company announced that the U.S. FDA has lifted the clinical hold on the Investigational New Drug (IND) application for DNL952, and company will proceed with the Phase 1 study.
  • 29 Jan 2026 According to a Denali Therapeutics Inc media release, data from the trial will be presented at the upcoming 22nd Annual WORLDSymposium to be held February 2-6, 2026, in San Diego, California.

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