STAT+: Ultragenyx reports early success with Angelman syndrome drug, backing up 2022 acquisition
STAT
APRIL 15, 2024
The biotech reported Monday that its drug, the antisense oligonucleotide treatment GTX-102, was generally safe and led to rapid and clinically meaningful improvements in cognition, communication, and sleep. The next step is a meeting with the Food and Drug Administration to discuss the findings and plans for a Phase 3 pivotal study.
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