The UBE3A-ATS antisense oligonucleotide rugonersen in children with Angelman syndrome: a phase 1 trial. - Hipp JF; Bacino CA; Bird LM; Bruenig-Traebert I; Chan D; de Wit MC; Fontoura P; Hooper G; Jagasia R; Krishnan ML; Murtagh L; Noci A; et al. (Nature medicine, 2025)