Shots:
- The US FDA has granted BTD to ION582 (UBE3A protein expression stimulant) for the treatment of Angelman syndrome
- Designation was based on P-I/II (HALOS) trial assessing ION582 in pts with Angelman syndrome, which showed improvements across all functional domains incl. communication, cognition & motor function; safety was favorable
- Additionally, ION582 is being investigated in a global P-III (REVEAL) trial assessing ION582 vs PBO in children and adults with AS who have a maternal UBE3A gene deletion or mutation
Ref: Ionis| Image: Ionis | Press Release
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