Trial Profile

An Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 in Participants With Angelman Syndrome

Status: Active, no longer recruiting

Phase of Trial: Phase I

Latest Information Update: 25 Jul 2025

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At a glance

11 Jul 2025 According to Oak Hill Bio media release, results from this study have been published on 11 July 2025 in Nature Medicine. Dr. Mark Shen, Assistant Professor of Neuroscience and Psychiatry at the University of North Carolina School of Medicine and one of the principal investigators in this trial was one of the authors of the paper.
11 Jul 2025 Results presented in the Oak Hill Bio Media Release.
15 Apr 2025 Results data from TANGELO study were presented at FAST's Annual Global Science Summit in November 2024.

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