Ultragenyx’s Angelman therapy fails Phase 3 trial in setback for
In a major setback to the search for a treatment for the rare neurodevelopmental disease known as Angelman syndrome, Ultragenyx said a Phase 3 trial of its experimental therapy failed. The company's antisense oligonucleotide therapy ...
September 2, 2026 09:35 PM EDT R&D Ultragenyx’s Angelman therapy fails Phase 3 trial in setback for company and patients Lei Lei Wu News Reporter In a major setback to the search for a treatment for the rare neurodevelopmental disease known as Angelman syndrome, Ultragenyx said a Phase 3 trial … Sign up to read this article for free. Get free access to a limited number of articles, plus choose newsletters to get straight to your inbox. SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER
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