TSHA-106 for the treatment of angelman syndrome; TSHA-114 for the treatment of fragile X syndrome; and TSHA-101 for the treatment of GM2 gangliosidosis. The company has research, collaboration ...

Fragile X syndrome, and Angelman syndrome. It also provides ANAVEX 3-71, which is in clinical trial for the treatment of schizophrenia, frontotemporal dementia, and Alzheimer’s disease.

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.

WASHINGTON _ Despite recent efforts to remove or rename Confederate memorials, there are still more than 2,000 symbols in places across the country, according to a report released Thursday.

Many say they felt attacked by debilitating waves of sound and pressure known as "Havana syndrome." Sunday, 60 Minutes reported… U.S. defense official had ‘Havana syndrome’ symptoms during a ...