RARE - Rare Disease-Focused-Ultragenyx Pharmaceutical Stock Falls On Serious Adverse Events In Eaarly Genetic Disorder Study | Benzinga
Monday, Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) revealed new data from the Phase 1/2 study of GTX-102 for Angelman syndrome, a genetic disorder causing developmental disabilities and nerve-related symptoms.
Patients in Expansion Cohorts A & B treated with a set dose and regimen of GTX-102 showed rapid and clinically meaningful improvement across multiple domains consistent with or exceeding Dose-escalation Cohorts 4-7 data at Day 170.
Treatment of the dose-escalation cohorts 4-7 showed long-term increasing and sustained clinical benefits far exceeding Natural History data at Day 758.
Expansion Cohorts at Day 170 on 24 patients:
- Cognition and receptive communication showed rapid and clinically significant improvement compared with Natural History data.
- Hyperactivity and noncompliance showed rapid and clinically significant improvement at Day 170 compared with Natural ...