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Aspire phase 3 study of GTX-102 in pediatric deletion-type Angelman syndrome
NL-OMON60152Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Feb 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
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Overview
Phase 3, single-arm trial of apazunersen in Angelman syndrome (AS), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoint: Change from baseline in Bayley-4 Cognitive Raw Score Without Caregiver Input at Day 338. Started Feb 2025; status: completed.
02
Drugs
apazunersen
03
Conditions
Angelman syndrome (AS)
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