Risdiplam
Risdiplam is an SMN2 splicing modifier designed to treat spinal muscular atrophy (SMA), a rare genetic neuromuscular disease caused by mutations in chromosome 5q that result in insufficient survival motor neuron (SMN) protein. By modifying the splicing of the SMN2 gene, risdiplam increases the production of functional SMN protein, helping to compensate for the defective SMN1 gene.
Risdiplam is an oral medication for spinal muscular atrophy that increases SMN protein production by modifying SMN2 gene splicing.
Key facts at a glance
- ATC
- M09AX10
- RxNorm
- 2390935
- Generic
- Risdiplam
- Class
- atc-m09
- Brands
- Evrysdi
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- tablet, solution
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Treatment of spinal muscular atrophy (SMA) in pediatric and adult patients, including Types 1, 2, and 3 SMA, caused by chromosome 5q mutations with biallelic SMN1 mutations.
Risdiplam is a small molecule that modifies the splicing of SMN2 pre-mRNA, promoting inclusion of exon 7 and increasing production of functional SMN protein to compensate for SMN1 deficiency.
Administered orally once daily with food. Requires regular monitoring of liver function, hematological parameters, and pregnancy status. Common side effects may include rash, diarrhea, nausea, and headache. Not recommended during pregnancy; adequate contraception required during treatment and after discontinuation.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy