Posted by Michael Wonder on 27 Jun 2025
Ultragenyx receives breakthrough therapy designation for GTX-102 in Angelman syndrome
27 June 2025 - Phase 3 Aspire study enrollment on track to complete in 2025.
Ultragenyx today announced that it has received breakthrough therapy designation from the US FDA for GTX-102 (apazunersen) as a treatment for Angelman syndrome.
