The first person has been dosed in a Phase 3 clinical trial testing GTX-102 (apazunersen), Ultragenyx Pharmaceutical’s experimental therapy, in…
Articles by Marisa Wexler, MS
The genetic medicine rugonersen (RO7248824) may be able to target the underlying cause of Angelman syndrome. A study found that…
The Phase 3 clinical trial that’s testing an experimental therapy for Angelman syndrome has finished enrolling patients. The…
The U.S. Food and Drug Administration (FDA) has given Mavrix Bio the go-ahead to start a first-in-human clinical trial testing…
Some people with Angelman syndrome may experience a phenomenon known as gelastic syncope, where laughter leads to a loss…
Rebecca Burdine, PhD, a professor at Princeton University, received two awards for her contributions to Angelman syndrome research. The…
Note: This story was updated Aug. 13, 2024, to correct the spelling of the measure of cognitive development to Bayley-4.
A molecule dubbed (S)-PHA533533 is able to increase the expression of the UBE3A protein in mouse and human cell models…
A previously unreported mutation in the UBE3A gene was found to be the cause of Angelman syndrome for two…
The Phase 1/2 KIK-AS clinical trial, which is testing the experimental genetic therapy GTX-102 in children with Angelman syndrome,…