Ultragenyx posts trial data backing Phase 3 design for Angelman syndrome therapy

seekingalpha

2024-11-10

Shidlovski

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced Saturday that GTX-102, its candidate for the rare neuro-genetic disorder Angelman syndrome, improved patient outcomes across multiple domains in a Phase 1/2 trial, supporting a Phase 3 trial design.

Citing Bayley-4 Cognition Growth Scale Value (GSV), an assessment

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