ZANVASTRO (zilganersen sodium)
ALEXANDER DISEASE
Ionis Pharmaceuticals, Inc.
Indication
Alexander disease
Alexander disease is a rare, progressive neurodegenerative disorder and primary astrogliopathy characterized by the abnormal accumulation of cytoplasmic protein aggregates known as Rosenthal fibers within astrocytes. Read more about Alexander disease
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Coming soonAlexander disease is a rare, progressive neurodegenerative disorder and primary astrogliopathy characterized by the abnormal accumulation of cytoplasmic protein aggregates known as Rosenthal fibers within astrocytes.
It predominantly damages the central nervous system white matter and is caused by gain-of-function variants in the *GFAP* gene, leading to defective intermediate filaments and demyelination.
The disease affects patients across the lifespan, classically manifesting in infants and young children with macrocephaly, seizures, spasticity, and severe psychomotor delay.
Later-onset juvenile and adult forms more prominently present with bulbar dysfunction, ataxia, palatal myoclonus, and autonomic deficits stemming from hindbrain involvement.
- medlineplus.gov
Alexander disease is a rare, progressive neurodegenerative disorder and primary astrogliopathy characterized by the abnormal accumulation of cytoplasmic protein aggregates known as Rosenthal fibers within astrocytes It predominantly damages the central nervous system white matter and is caused by gain-of-function variants in the *GFAP* gene, leading to defective intermediate filaments and demyelination The disease affects patients across the lifespan, classically manifesting in infants and young...
- nih.gov
Alexander disease is a rare, progressive neurodegenerative disorder and primary astrogliopathy characterized by the abnormal accumulation of cytoplasmic protein aggregates known as Rosenthal fibers within astrocytes It predominantly damages the central nervous system white matter and is caused by gain-of-function variants in the *GFAP* gene, leading to defective intermediate filaments and demyelination The disease affects patients across the lifespan, classically manifesting in infants and young...
- rarediseases.org
Alexander disease is a rare, progressive neurodegenerative disorder and primary astrogliopathy characterized by the abnormal accumulation of cytoplasmic protein aggregates known as Rosenthal fibers within astrocytes
- medlineplus.gov
It predominantly damages the central nervous system white matter and is caused by gain-of-function variants in the *GFAP* gene, leading to defective intermediate filaments and demyelination
- chop.edu
The disease affects patients across the lifespan, classically manifesting in infants and young children with macrocephaly, seizures, spasticity, and severe psychomotor delay
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