News

Treating Sleep, GI Problems May Help Ease Behavioral Challenges

Most children and adolescents with Angelman syndrome experience problems with sleep and digestive issues such as constipation, a new study highlights. Challenging behaviors such as aggression are generally more common and more severe in these children, which suggests that treating underlying symptoms may help to manage their behaviors. “Findings…

Trial: Gene Therapy GTX-102 Shows Promise in Angelman Children

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…

Phase 2 Trial of NNZ-2591 for Angelman Opens in Australia

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…

Study: Seizure Control May Benefit Development in Angelman Children

Most children with Angelman syndrome experience substantially delayed development, a new study from China highlights. Such developmental delays are especially pronounced in Angelman patients who experience seizures — which implies that seizure control may be beneficial for the development of children with the neurological disorder. The findings indicated…

FDA Acts to Support Development of Ionis Therapy ION582

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…