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

The now-21-year-old has Angelman syndrome, and the actor called his ... He called sobriety his “legacy,” explaining, “Generations of people have had their lives ruled and ruined by ...

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

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 ...

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 Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the pivotal Phase 3 study design following successful alignment with the U.S. Food and Drug Administration (FDA) on ION582, an ...

Angelman syndrome is a severe and lifelong neurodevelopmental disorder that is estimated to affect approximately 1 in 12,000 to 1 in 20,000 people globally. It is caused by loss of function in the ...

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 ...

(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 ...

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 ...

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 ...