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Learn About the Neurologic Diseases in Our Pipeline

The neurology pipeline includes investigational RNA-targeted medicines for severe and debilitating neurologic diseases that affect a wide range of people.

If you are interested in more disease state education, please download the appropriate information sheet below:

Angelman Syndrome (AS) disease state education download

Angelman Syndrome (AS)


Pediatric Neurology disease state education download

Pediatric Neurology (Alexander Disease, MECP2 Duplication Syndrome, Pelizaeus-Merzbacher Disease)


Fused in Sarcoma Amyotrophic Lateral Sclerosis (FUS-ALS) disease state education download

Fused in Sarcoma Amyotrophic Lateral Sclerosis (FUS-ALS)


Prion Disease (PrD)disease state education download

Prion Disease (PrD)


Multiple System Atrophy (MSA) disease state education download

Multiple System Atrophy (MSA)


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