GTX-102, a gene therapy for Angelman syndrome, continues in a clinical trial to be safe and show benefit when…
Articles by Margarida Maia, PhD
The diuretic bumetanide helped to improve cognition and lengthen the time to a seizure in a mouse model of…
The U.S.-based Angelman Syndrome Foundation (ASF) and the Canadian Angelman Syndrome Society (CASS) have partnered up to broaden their…
Breaking into a smile may hold the attention of people with Angelman syndrome (AS) for longer than just holding…
GeneTx Biotherapeutics, a company set up by the Foundation for Angelman Syndrome Therapeutics (FAST), has been acquired by…
Researchers brought a message of hope for the Angelman syndrome community as they discussed advances in treatment and scientific…
A first patient in the U.K. has been dosed in the resumption of a Phase 1/2 clinical trial evaluating the…
Four foundations are collaborating to fund the addition of Angelman syndrome and other conditions caused by changes on chromosome 15 to …
The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on a Phase 1/2 study testing GTX-102 —…
Neuren Pharmaceuticals is a step closer to Phase 2 clinical testing of NNZ-2591, its investigational therapy for…