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Does Evrysdi cure spinal muscular atrophy (SMA)?

Medically reviewed by Carmen Fookes, BPharm. Last updated on Sep 7, 2020.

Official Answer

by Drugs.com

Evrysdi does not cure spinal muscular atrophy (SMA), it just replaces a protein that is low or missing in children or adults with the condition, improving muscle strength and allowing some relief from some of the symptoms of SMA. Evrysdi must be taken for a person’s lifetime; if Evrysdi is stopped the child or adult’s SMA symptoms will progressively worsen at a faster rate.

How does Evrysdi work?

Evrysdi (risdiplam) works by targeting the SMN2 gene (survival motor neuron gene 2), causing it to make more functional SMN protein. This increases SMN protein levels throughout the central nervous system and body, helping to improve motor nerve and muscle function in children and adults with SMA.

Evrysdi was approved by the FDA on August 7, 2020) and is available as a liquid that is given by mouth or through a gastrostomy tube (g-tube) once a day after a meal. It may be used to treat all types of spinal muscular atrophy (SMA) in adults and children aged 2 months of age and older.

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