Evrysdi (Risdiplam) for SMA

Overview

Evrysdi is a prescription medication approved by the U.S. Food and Drug Administration (FDA) to treat children and adults with spinal muscular atrophy (SMA). Evrysdi is also known by its drug name, Risdiplam.

Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier. It is believed to increase the production of full-length SMN, a protein which is deficient in people with SMA due to an inactivating gene mutation. SMN normally functions in important cellular processes, including DNA to RNA conversion and moving materials into and out of cells.

How do I take it?

Evrysdi is available as a powder that is mixed to produce an oral solution.

Evrysdi is taken orally once a day after a meal using an oral syringe.

Side effects

The FDA-approved label for Evrysdi lists common side effects experienced by people with later-onset SMA, including fever, diarrhea, and rash. For those with infantile-onset SMA, common side effects include fever, diarrhea, rash, upper respiratory tract infection, pneumonia, constipation, and vomiting.

If you are pregnant or planning to become pregnant, consult your doctor before taking Evrysdi, as this drug may cause fetal harm.

For more details about this treatment, visit:

Evrysdi — Genentech