Angelman syndrome, which affects 1 in 15,000 people ... so you’re left with a young adult who should be an integrated part of ...

Colin Farrell rarely opens up about his private life, but in recent times he's made an exception to honour his beloved son ...

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 ...

Ionis has announced the design for a Phase III trial of ION582, a treatment for Angelman syndrome (AS), following discussions with the FDA.

Phase 1/2 data show improvements across all domains and confirm that Phase 3 Aspire study is amply powered to establish efficacy of GTX-102Phase ...

(RARE) announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome. The Phase 1/2 data showed improvements ...

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 ...

The Phase 3 trial, dubbed the REVEAL study, aims to enroll roughly 200 children and adults with AS ... including ION582 for Angelman syndrome. However, it's important to note that Ionis is ...

There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome, suggesting that improvement ...

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 ...

Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in ...