Dysautonomic Phenotype in Male Patients With MECP2 Mutation

计划入组 20 人 · 首次发布于 2020 年 8 月 6 日

Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome

计划入组 8 人 · 首次发布于 2025 年 3 月 4 日

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

MECP2 targeting antisense oligonucleotides

MECP2 duplication syndrome (Cholgene)

MECP2-ASO(Baylor College of Medicine)

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

Engineered Toxoplasma Gondii Delivers Therapeutic Proteins to Mouse Brain

Two Scientists Win 2026 Horwitz Prize for Rett Syndrome Breakthrough