Zilganersen (Zanvastro)

作用机制

Zanvastro is an antisense oligonucleotide (ASO) that works by reducing glial fibrillary acidic protein (GFAP), the toxic accumulation of astrocytes in the brain that leads to the creation of Rosenthal fibers which disrupt neurological function.

适应症

Zanvastro (zilganersen) is indicated for the treatment of pediatric and adult patients with Alexander disease, a rare, progressive and often fatal neurological disorder that can affect motor, cognitive, autonomic and gastrointestinal function.

• Alexander disease