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Phase 3 study of ION582 in children and adults with Angelman syndrome
MOH_2026-02-15_015658Phase 3Recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Jun 2025
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of obudanersen in Angelman syndrome (AS) (4 cohorts), sponsored by Ionis Pharmaceuticals. Primary endpoint: Expressive communication at Week 52. Started Jun 2025; status: recruiting.
02
Drugs
obudanersen
03
Conditions
Angelman syndrome (AS)
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