Lila Levinson, PhD,  Science Writer—

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…

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…