Ultragenyx Reveal New Data From Phase 1/2 Study Of GTX-102 For Treatment Of Angelman Syndrome
Author: Benzinga Newsdesk | April 15, 2024 07:07am
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 benefit far exceeding Natural History data at Day 758.
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