News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to MVX-220, Mavrix Bio’s investigational gene therapy for Angelman syndrome that is headed into clinical trials. Fast track designation aims to facilitate the clinical development and regulatory review of therapies intended to treat serious conditions with an…

The genetic medicine rugonersen (RO7248824) may be able to target the underlying cause of Angelman syndrome. A study found that the treatment — tested in early clinical trials and expected to enter Phase 3 trials early next year — restored UBE3A protein activity in mouse and primate models of the…

The Phase 3 clinical trial that’s testing an experimental therapy for Angelman syndrome has finished enrolling patients. The Aspire study (NCT06617429), whose results are expected in late 2026, achieved full enrollment, with about 129 participants, barely half a year after it began enrolling last December, according to…

The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to GTX-102 (apazunersen), a gene therapy being developed by Ultragenyx Pharmaceutical for treating Angelman syndrome. “FDA breakthrough therapy designation underscores both the urgent need for an effective treatment for patients and families affected by Angelman syndrome…

The U.S. Food and Drug Administration (FDA) has given Mavrix Bio the go-ahead to start a first-in-human clinical trial testing MVX-220, the company’s experimental gene therapy for Angelman syndrome. The FDA approved Mavrix’s investigational new drug application (IND), which is a formal request to begin clinical trials of the…

A diet supplemented with octanoic acid safely improved behavior across multiple tests in a mouse model of Angelman syndrome, a study reports. A fatty acid found naturally in breast milk, octanoic acid helped reactivate a gene that’s normally suppressed in the brain. This led to improvements in memory and…

Sensory problems and other features of autism spectrum disorder (ASD) were prevalent in children with Angelman syndrome and were generally stable over time, a report suggests. Children with the most common type of genetic defect that causes Angelman, called the deletion genotype, exhibited more autistic traits than those…

Some people with Angelman syndrome may experience a phenomenon known as gelastic syncope, where laughter leads to a loss of consciousness, a report involving a teenager and two adults highlights. “The goal of this article is to bring specific awareness of gelastic syncope to clinicians and caregivers and to…

Supporters are gearing up for International Angelman Day (IAD), a community-driven event held every February that aims to raise awareness for Angelman syndrome and honor those living with or lost to the rare neurodevelopmental disorder. The celebration also aims to promote Angelman research and education and mobilize fundraising…

The first patient has been dosed in a Phase 3 trial evaluating the safety and efficacy of GTX-102, Ultragenyx Pharmaceutical’s experimental gene therapy for Angelman syndrome. The global Aspire study (NCT06617429) is expected to enroll about 120 children and adolescents, ages 4 to 17, with Angelman…