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

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.

Symptoms of Werner syndrome, which causes premature aging, can appear in a person's teens and progress quickly in their 20s ...

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

While people who have a family history of type 1 diabetes may be predisposed to developing it, the inheritance pattern in most cases is unclear. Type 2 diabetes results when the body doesn’t use ...