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

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

When she was a baby, Scottie Mae Blair was diagnosed with Angelman syndrome—a rare genetic condition that causes severe physical and learning disabilities. Her parents were told to expect that their ...

Actor Colin Farrell is best known for his roles on the big screen, but his roles at home including that of father to his two ...

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 ...

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 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 ...

A recent breakthrough in genetic research has introduced a tool capable of delivering genetic material to developing fetal ...

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

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

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

Overall, 97% of people in the medium and high dose groups assessed in the study saw an improvement in overall AS symptoms as measured by the Angelman Syndrome Clinical Global Improvement Change ...