Ultragenyx Pharmaceutical Inc. (RARE) announced Phase 1/2 data in support of the Phase 3 Aspire study for GTX-102, its ...

14 Nov – Fred Cheng recently celebrated turning 41 by revealing that his focus this year has been on son Asher.

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

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

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced Saturday that GTX-102, its candidate for the rare neuro-genetic disorder ...

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 ...

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

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 ...

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 ...

Joseph Schwartz has given his Buy rating due to a combination of factors surrounding Ultragenyx Pharmaceutical’s GTX-102 program for Angelman syndrome. The updated data from the Phase 1/2 trial, ...

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 ...

James Cronin is taking part in the world’s most famous marathon next April and is pledging to raise £10,000 for a charity, ...