News

Anxiety, repetitive behaviors predict sleep problems in Angelman

Anxiety and repetitive behaviors are significant predictors of sleep problems among children and adolescents with Angelman syndrome, according to a new study using global registry data. Language and communication difficulties and challenging behaviors also were linked to sleep problems in these patients. “Future research needs to replicate this research…

Angelman syndrome treatment ION582 eases symptoms: Trial data

Ionis Pharmaceuticals’ investigational Angelman syndrome treatment ION582 demonstrated a favorable safety profile and lessened overall symptoms in patients participating in the HALOS clinical trial. That’s according to six-month follow-up data from part one of Phase 1/2 study (NCT05127226), which tested three doses of ION582 — low, medium and…

Translating Angelman patient registry boosted participation

The translation into other languages of the Global Angelman Syndrome Registry (GASR) website has substantially boosted global participation by families affected by Angelman syndrome, a study shows. Researchers say adopting multiple languages “helps build and maintain engagement with global disease communities.” The approach could also serve as an…

Results of KIK-AS Angelman gene therapy study due 1st half of year

The Phase 1/2 KIK-AS clinical trial, which is testing the experimental genetic therapy GTX-102 in children with Angelman syndrome, has finished enrolling patients. With 74 enrolled, including 53 in dose-expansion groups, the therapy’s developer Ultragenyx Pharmaceutical said results will be available in the next few months. “With completion…