Angelman syndrome is caused by a large deletion of the region of the maternal chromosome that contains UBE3A, or by a DNA sequence change (mutation) in the UBE3A gene inherited from the mother.

For the first time, Colin Farrell has opened up his home and talked in-depth about life with his son James, who has Angelman ...

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 ...

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 ...

chief scientific officer at the Foundation for Angelman Syndrome Therapeutics. Angelman syndrome is a neurogenetic disorder that occurs in 1 in 15,000 births.

(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 ...

Considering taking supplements to treat angelman syndrome? Below is a list of common natural remedies used to treat or reduce the symptoms of angelman syndrome. Follow the links to read common ...

NOVATO, Calif., Nov. 09, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational ...

(NASDAQ: RARE) today announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its investigational antisense oligonucleotide for Angelman syndrome, that will be presented at the ...