Ionis Pharmaceuticals Inc.

Research activity includes amyotrophic lateral sclerosis with FUS mutations, Alexander disease, Angelman syndrome, Pelizaeus-Merzbacher disease, multiple system atrophy, and prion disease. The portfolio also includes studies in MECP2 duplication syndrome and Alzheimer’s disease in individuals with Down syndrome.

• FUS-ALS
• Angelman syndrome
• Multiple system atrophy
• Prion disease
• MECP2 duplication syndrome

These programmes reflect sustained interest in rare inherited and progressive conditions affecting the central nervous system.