He had a genetic condition called Alexander disease, and the outlook was bleak. Immediately the geneticist said, “At the moment, there is no treatment, no cure and life expectancy is five to 10 ...

Alexander disease (AxD) is a rare condition affecting the central nervous system (CNS). It belongs to a group of disorders known as leukodystrophies, which involve the destruction of myelin ...

Zilganersen is the first investigational medicine in clinical development for people with Alexander disease, a rare, life-threatening neurological condition ...

MADISON - Scientists have pinpointed the gene responsible for a rare and devastating childhood brain disorder called Alexander disease, solving a 50-year-old mystery regarding its cause.

Zilganersen is an investigational antisense oligonucleotide designed to stop excess GFAP production. The Food and Drug Administration (FDA) has granted Fast Track designation to zilganersen for ...

Zilganersen is the first investigational medicine in clinical development for adults and children living with Alexander disease, an ultra-rare neurological condition "With no approved treatments ...

Alexander disease is a leukodystrophy that is neuropathologically characterized by the presence of numerous Rosenthal fibers in astrocytes. Recently, mutations in the gene encoding glial ...

AxD is estimated to occur in an estimated one in one million people in the U.S. and can present throughout life. 1-3 The disease is a result of genetic variants in the glial fibrillary acidic ...

Zilganersen is the first investigational medicine in clinical development for adults and children living with Alexander disease, an ultra-rare neurological condition ...