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, 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 ...

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 efficacy of GTX-102Phase ...

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

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.

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

Colin Farrell is doing his bit to help families with adult children with disabilities like his own son to get the support ...

Colin Farrell is doing his bit to help families with adult children with disabilities like his own son to get the support they need.

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 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 ...