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

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.