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.
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.