Ionis' Rare Development Disorder Drug Candidate 'Could Be Differentiated' From Peer Ultragenyx: Analyst

Loading... Ionis Pharmaceuticals Inc IONS released results Monday from the completed multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582 in people with Angelman syndrome (AS). AS is a rare neurodevelopmental disorder caused by a loss of function in the maternal UBE3A gene. It affects an estimated 1 in 21,000 people worldwide. The data demonstrated consistent and encouraging clinical improvement on assessing all functional domains, including communication, cognition, and motor f ...