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A Phase 1-2, Double-Blind, Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients With MECP2 Duplication Syndrome

The primary purpose of this study is to evaluate the safety and tolerability of ION440.

Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome

Ionis Pharmaceuticals, Inc.NCT06430385

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