Fred Cheng recently celebrated turning 41 by revealing that his focus this year has been on son Asher. On 11 November, the ...

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, ...

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

A four-year-old girl with DUP15q syndrome, who doctors said would never walk, has defied expectations to take her first steps.

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

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

Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, announced today that it will present new preclinical ...

Ionis has announced the design for a Phase III trial of ION582, a treatment for Angelman syndrome (AS), following discussions with the FDA.

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

Off a rural road in Medina County is a little church in Valley City where a women’s ministry is having a big impact far beyond the four walls of their church.

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 ...

ASHG welcomed eight 2024–2026 Human Genetics Scholars who are recognized for their accomplishments and commitment to fostering DEI in human genetics and genomics.