Evrysdi (risdiplam) is a disease-modifying therapy used to treat spinal muscular atrophy (SMA) in adults and children 2 months of age and older.
How effective is Evrysdi (risdiplam) for SMA?
The approval of Evrysdi (risdiplam) was based on 2 main studies in more than 450 people with SMA aged 2 months to 25 years.
According to the study results published:
Type 1 SMA
Evrysdi helped infants achieve a key motor milestone:
Evrysdi (risdiplam) helped infants survive without permanent breathing support:
Type 2 and 3 SMA
Motor neuron function improved in children and adults.
Evrysdi increased motor function with 1.6 points, as measured using the MFM32 scale. The score increased 1.36 points for those taking Evrysdi compared with a 0.19-point decrease for those not taking Evrysdi.
Which side effects can I expect with Evrysdi (risdiplam)?
The most common side effects of Evrysdi (which may affect more than 1 in 10 people) in people with SMA listed in the official prescribing information are fever, diarrhea, rash, runny nose, sneezing, sore throat, and cough (upper respiratory infection), lung infection, constipation and vomiting.
Please note this is not intended to be a comprehensive guide. Consult your treating doctor and the prescribing information at the bottom of the page for full details of side effects.
How is Evrysdi (risdiplam) taken?
Administration: take Evrysdi once a day (at the same time every day) by mouth or feeding tube.
Dosage: your healthcare provider will determine the right dose for you.
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