Ionis Pharmaceuticals has announced the design for a pivotal Phase III trial of ION582, an investigational treatment for AS, ...

Ultragenyx Pharmaceutical (RARE) shows promising results for GTX-102 in Angelman syndrome Phase 1/2 trial, supporting a Phase ...

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.

Encouraged by the technology’s performance, they speculated that their LNP technology could be effective not just against Angelman syndrome, but against other genetic-based neurodevelopmental ...

The REVEAL trial aims to address the issues of the patients living with this genetic disorder. "Ionis announces design for Phase III trial of Angelman syndrome therapy" was originally created and ...

It has applications in vaccine development, gene editing and protein replacement therapy. Recently ... their tool on the gene responsible for Angelman syndrome, a rare neurodevelopmental condition.

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

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 ...