The symptoms of SMA are caused by SMN protein deficits

SMA is caused by insufficient levels of functional SMN protein throughout the body 2

People with SMA may face severe consequences

In infantile-onset disease, most patients are not expected to survive

In later-onset disease, children and adults may face progressive loss of function

Proposed MOA

Evrysdi, an oral SMN2-splicing modifier, is designed to address the cause of SMA 1

  • Based on preclinical studies, Evrysdi was shown to increase exon 7 inclusion in SMN2 mRNA transcripts 1
    • This led to an increased production of full-length SMN protein in the brain
    • Preclinical data also indicates Evrysdi may cause alternative splicing of other genes, including FOXM1 and MADD

Evrysdi led to an increase in SMN protein

In clinical trials, treatment increased and sustained functional SMN protein levels 1

CNS=central nervous system; MOA=mechanism of action; SMN=survival motor neuron.

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