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

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 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.

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

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 ...

Scottie Mae Blair was diagnosed with Angelman syndrome at 11-months-old. The rare genetic condition causes severe physical and learning disabilities. Upon the diagnosis, parents Logan Blair, 32, and ...