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 work by increasing the production of SMN, a protein needed for muscle function. People with SMA have low SMN levels due to a gene mutation (change). This protein helps with essential cell functions, like processing genetic instructions and moving materials in and out of cells.

How do I take it?
Prescribing information states that Evrysdi is taken once daily with or without food. It is available in two forms:

• Oral solution — A powder that must be mixed by a healthcare provider before dispensing and that is taken using an oral syringe
• Tablet — A pill that must be swallowed whole with water or dissolved in filtered (nonchlorinated) water before taking

The dosage is based on age and body weight. Evrysdi should be taken exactly as prescribed by a healthcare provider.

Side effects
Common side effects in people with later-onset SMA include fever, diarrhea, and rash. In infants with SMA, additional side effects may include respiratory infections, constipation, vomiting, and cough.

Serious but rare side effects may include severe respiratory infections and difficulty swallowing. Evrysdi may also harm an unborn baby, so people who are pregnant or planning to become pregnant should talk to their doctor before using it.

For more information about this treatment, visit:

Prescribing Information: Evrysdi (Risdiplam) — Genentech