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.
Ionis Receives Breakthrough Therapy Designation for ION582 in Angelman Syndrome
Ionis Pharmaceuticals’ ION582 has received FDA Breakthrough Therapy Designation for Angelman syndrome, a status reserved for drugs showing substantial improvement over existing treatments.
First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman syndrome
Ionis Pharmaceuticals has dosed the first participant in its global Phase 3 REVEAL trial, evaluating the safety and efficacy of ION582 as a potential treatment for Angelman syndrome.
MavriX Bio Announces FDA Clearance of IND Application to Initiate First-in-Human Study of Gene Therapy for Angelman Syndrome
The FDA has cleared MavriX Bio’s IND application for MVX-220, an investigational AAV gene therapy for Angelman syndrome.