Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

计划入组 29 人 · 首次发布于 2023 年 8 月 28 日

A First-in-Human Clinical Trial to Evaluate the Safety, Tolerability, and Efficacy of a Novel CRISPR RNA-editing Therapy in Patients with Mecp2 Duplication Syndrome, a Rare Orphan Disease (HERO)

计划入组 6 人 · 首次发布于 2024 年 9 月 26 日

ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)

计划入组 48 人 · 首次发布于 2024 年 5 月 28 日

MECP2 duplication syndrome (Cholgene)

AAV9-MeCP2 gene therapy (rett syndrome)

MECP2 targeting antisense oligonucleotides

ALPORT SYNDROME FOUNDATION

United States

International Rett Syndrome Foundation

United States

RETT SYNDROME RESEARCH TRUST

United States

Gene Therapies Show Promise in Targeting the Root Cause of Rett Syndrome

Researchers Identify Novel Therapeutic Strategy for Rett Syndrome Through Alternative Splicing Modulation

RNA Editing Breakthroughs Show Clinical Promise with Wave Life Sciences Leading First Human Results