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 ...

Researchers have developed a novel delivery system for sending CRISPR reagents to brain cells, where it performs its gene ...

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced Saturday that GTX-102, its candidate for the rare neuro-genetic disorder Angelman syndrome, improved patient outcomes across multiple domains in a ...

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 ...