Patricia Inacio, PhD,  science writer—

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…

Ultragenyx Pharmaceutical has been cleared by the U.S. Food and Drug Administration to proceed to higher dosing of its investigational gene therapy GTX-102 in children and adolescents with Angelman syndrome in a Phase 1/2 clinical trial. The amendment to the KIK-AS trial (NCT04259281) protocol in the…

A tailored behavioral intervention program led to longer sleep time, better sleep habits, and improved quality of life in children with Angelman syndrome, a Dutch study reports. The findings suggest “behavioral intervention has a positive and persistent effect on sleep problems in children with AS [Angelman symdrome],” the researchers…

A history of reflux and night sweats during infancy may help identify children and adolescents with Angelman syndrome who are at higher risk for more gastrointestinal (GI) symptoms, according to a new study. “GI symptoms were found to be very common in people with AS and significant associations were…

The Foundation for Angelman Syndrome Therapeutics (FAST) has awarded a $500,000 grant to support the development of a less-invasive, brain-directed therapy for people with Angelman syndrome. The two-year grant was awarded to Barbara Bailus, PhD, an assistant professor of genetics at Keck Graduate Institute, in California. Her winning…

The investigational gene therapy GTX-102 shows potential to lessen disease severity and improve function in children and adolescents with Angelman syndrome, according to interim data from a Phase 1/2 clinical trial. Six-month follow-up data for 11 patients showed that treatment with GTX-102 resulted in improvements across exploratory…

A Phase 2 trial of NNZ-2591, Neuren Pharmaceuticals’ investigational oral therapy for Angelman syndrome, has started recruiting pediatric patients in Australia. The Australian study — which was also given regulatory clearance in the U.S. — will be conducted at three hospitals in that country: one each in Queensland, New…

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease status to ION582, Ionis Pharmaceutical’s investigational treatment for Angelman syndrome. The announcement of the new FDA designations — both aimed at encouraging the therapy’s development — follows the launch of the…

A diet enriched with a nutritional formula supplemented with ketones — natural metabolites that arise from the breakdown of fat — may improve fine motor skills, memory, and cognition in children with Angelman syndrome, according to the results from a small trial. Funded by the Foundation for Angelman…

Epileptic seizures occur earlier and more frequently, leading to more hospitalizations, in children with Angelman syndrome due to a missing UBE3A gene, according to a retrospective analysis of a natural history study. The study, “Clinical Characterization of Epilepsy in Children With Angelman Syndrome,” was published in the journal…