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Trial intelligence / Profile preview
Open-label phase 1/2 dose-escalation and multiple-dose study of GTX-102 in pediatric Angelman syndrome
MOH_2023-03-08_012404Phase 2Recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Jan 2023
- 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, open-label, single-arm trial of apazunersen in Angelman syndrome (AS), sponsored by Ultragenyx Pharmaceutical Inc. Started Jan 2023; status: recruiting.
02
Drugs
apazunersen
03
Conditions
Angelman syndrome (AS)
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