Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish ...

New preclinical data for candidate ETX201 illustrate its potential as a first-in-class gene therapy for Angelman syndrome. Studies in non-human primates demonstrate safety, biodistribution ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

The technology, tested in mice, might have the potential to stop the progression of genetic-based neurodevelopmental conditions, such as Angelman syndrome and Rett syndrome before birth.

The technology, tested in mice, might have the potential to stop the progression of genetic-based neurodevelopmental conditions, such as Angelman syndrome and Rett syndrome, before birth.

A four-year-old girl with DUP15q syndrome, who doctors said would never walk, has defied expectations to take her first steps.

14 Nov – Fred Cheng recently celebrated turning 41 by revealing that his focus this year has been on son Asher.

A four-year-old girl who doctors said would never walk has defied the odds and taken her first steps. Scottie Mae Blair was ...

The global Phase 3 Aspire study will enroll approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion and will include a 48-week ...

The global Phase 3 Aspire study will enroll approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion and will include a 48-week ...

Colin Farrell’s son James had a part to play in his sobriety journey. The now-21-year-old has Angelman syndrome, and the actor called his child’s diagnosis “fuel” in choosing to abstain fr ...