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

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

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

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

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

New preclinical data for candidate ETX201 illustrate its potential as a first-in-class gene therapy for Angelman syndrome. Studies in non-human primates demonstrate safety, biodistribution ...

Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the drug.

Colin Farrell’s son James had a part to play in his sobriety journey. The now-21-year-old has Angelman syndrome, and the actor called his child’s diagnosis “fuel” in choosing to abstain fr ...

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 ...

She’s made her name counting dollars and cents for tens of thousands of Australians, helping to manage their budgets and ...