Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome
The Phase 3 ASPIRE study for GTX-102 (apazunersen) did not meet its primary or key secondary endpoints. FAST expresses gratitude to participating families and awaits further details from Ultragenyx.
Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome
Ultragenyx’s GTX-102 (apazunersen) has received FDA Breakthrough Therapy Designation for Angelman syndrome, marking a key milestone in its path toward potential approval.