Ultragenyx's Phase 3 Aspire trial of apazunersen misses its primary and key secondary endpoints in Angelman syndrome, sending shares down more than 40%.
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 …
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The failure of what was once considered a potential blockbuster therapy represents a major setback in a tough-to-treat disease…
The company's experimental Angelman syndrome treatment failed.
Oak Hill Bio, a new company backed by RA Capital and other investors, will take on Ultragenyx and Ionis in developing an Angelman…
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CAMP4 is preparing a Phase 1/2 trial of an experimental SYNGAP1 therapy, marking a major milestone for patients with the rare…
