Background/aim Prader-Willi syndrome (PWS) and Angelman syndrome (AS) are rare imprinting disorders caused by the aberrant ...

Ionis Pharmaceuticals has dosed the first subject in the global, randomised Phase III REVEAL trial of investigational ION582, aimed at treating individuals with Angelman syndrome (AS), a rare ...

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.

The firm expects to complete enrolling the REVEAL trial, in which patients will receive the ASO, dubbed ION582, in 2026.

Apr. 15, 2025 — Certain common genetic changes might make some people with focal epilepsy less responsive to seizure medications, finds a new global ... Could an Arthritis Drug Unlock Lasting ...

Turner syndrome is a rare genetic disorder found in some women and people assigned female at birth. It can cause short stature and a range of health issues including heart defects, learning ...