How Does Evrysdi Treatment Work?
Evrysdi, also known as risdiplam, is a disease-modifying SMA treatment designed to help the body make more functional SMN protein by targeting the backup SMN2 gene. The FDA prescribing information describes Evrysdi as a survival motor neuron 2, or SMN2, splicing modifier indicated for the treatment of Spinal Muscular Atrophy in pediatric and adult patients.
Evrysdi works by modifying how the SMN2 gene is processed so the body can include more exon 7 in SMN2 messenger RNA, which helps increase production of full-length SMN protein. In simpler terms, Evrysdi does not replace the missing or nonworking SMN1 gene; instead, it helps the body use its backup gene more effectively. FDA labeling explains that risdiplam increased exon 7 inclusion in SMN2 mRNA transcripts and increased full-length SMN protein production in the brain during laboratory and animal model studies. Evrysdi is an FDA-approved daily medication for pediatric and adult patients of all ages and types of SMA.
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