Mecp2 Duplication Syndrome
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CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
An observational trial to characterize biomarkers and disease progression in MECP2 duplication syndrome sponsored by Ionis Pharmaceuticals h…
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: 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) [Phase 1, Phase 2]
This is a Phase 1/2 trial evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of intrathecally administered ION440 i…
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CLINICAL_TRIALS research · 2024-11-26Clinical Trial Update: 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) [NA]
A first-in-human clinical trial to evaluate a novel CRISPR RNA-editing therapy for MeCP2 duplication syndrome has begun, with HuidaGene Ther…
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CLINICAL_TRIALS trial_result · 2017-03-16Clinical Trial Update: Genetic and Physical Characteristics of Rett Syndrome
A completed clinical trial update examining the genetic and physical characteristics of Rett syndrome, including MECP2 duplication syndrome.