Ultragenyx’s Angelman therapy fails Phase 3 trial in setback for
Phase 3negativeNegative
AI Analysis
Summary
Ultragenyx's Phase 3 trial of an antisense oligonucleotide therapy for Angelman syndrome has failed, representing a significant setback in the development of treatments for this rare neurodevelopmental disease.
Clinical Trial Data
Phase
3
Primary Endpoint
Not Met
Outcome Details
Phase 3 trial failed
Importance:8/10
Sentiment:
-0.90
Phase 3 failureAngelman syndromeAntisense oligonucleotideRare diseaseClinical trial
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Published by Endpoints News on September 3, 2026 1:35 AM