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

Ultragenyx Pharmaceutical (RARE) shows promising results for GTX-102 in Angelman syndrome Phase 1/2 trial, supporting a Phase ...

A four-year-old girl, who doctors said would never walk, has defied expectations to take her first steps. When she was a baby, Scottie Mae Blair was diagnosed with Angelman syndrome—a rare genetic ...

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 ...

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 ...

Ionis Pharmaceuticals has announced the design for a pivotal Phase III trial of ION582, an investigational treatment for AS, ...

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

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

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 ...

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

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) has confirmed plans to initiate a Phase 3 study for ION582, a potential treatment for Angelman syndrome (AS), in the first half of 2025. The decision ...