Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
1
Pipeline Programs
1
Companies
1
Clinical Trials
1 recruiting
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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1
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2024
2025
2026
2027
2028
2029
2030
Ionis PharmaceuticalsION440
Clinical Trials (1)
Total enrollment: 48 patients across 1 trials
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)
Start: Oct 2024Est. completion: Apr 203048 patients
Phase 1/2Recruiting
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 actively recruiting trials targeting 48 patients
1 companies competing in this space