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, ...

Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...

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 ...

What is staphylococcal scalded skin syndrome in children? Staphylococcal scalded skin syndrome (SSSS) is a serious skin infection. The infection causes peeling skin over large parts of the body. It ...

He hopes to raise £10,000 for the charity, which supports those affected by Angelman syndrome, a rare neurological condition. Read more: Waddle of Worcester auction raises thousands for St ...

The technology, tested in mice, might have the potential to stop the progression of genetic-based neurodevelopmental conditions, such as Angelman syndrome and Rett syndrome before birth.

The Science Corp study, dubbed PRIMAvera, examined 38 patients suffering from geographic atrophy (GA), who were implanted with the PRIMA retinal system. Credit: Shutterstock / metamorworks US-based ...