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Phase 2 open-label basket study of GTX-102 in children and adults with Angelman syndrome
MOH_2026-02-02_015633Phase 2Not yet recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Jan 2026
- 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 2, randomised, open-label trial of apazunersen in Angelman syndrome (AS) (3 arms), sponsored by Ultragenyx Pharmaceutical Inc. Planned start Jan 2026; status: not yet recruiting.
02
Drugs
apazunersen
03
Conditions
Angelman syndrome (AS)
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