Angelman syndrome is a rare neurodevelopmental disorder characterized by changes in brain structure, severe intellectual disability, impairments in speech, motor function, epilepsy, sleep, and unique ...

Part 1 of HALOS trial evaluating ION582 has completed enrollment with results expected in mid-2024 ION582 has been generally well tolerated at all dose levels, and participants are continuing to Part ...

Angelman syndrome affects roughly 1 in every 20,000 children and it has no approved treatment. Researchers recently published results of early tests of a gene therapy strategy for Angelman syndrome, a ...

Data from the TANGELO trial provides evidence of improvement in brain activity and developmental ability in children with Angelman syndrome compared to natural history Angelman syndrome is caused by ...

All of 2-year-old Grayson Jacob newborn screenings and in-utero ultrasounds were normal, yet at 6 weeks old the Austin boy was given a "failure to thrive" label. He had acid reflux, wasn't growing and ...

Researchers in Purdue University’s College of Health and Human Sciences rely on human subjects for learning more about genetic and developmental disorders like autism, Angelman syndrome and others.

Henry Edberg dug through the play kitchen at his home in Rosemount before serving up the daily special to his older brother. “Vegetables are healthy, so I’m giving you the vegetables,” Henry, 3, told ...

Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that ...

Scientists have reported encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat ...