Risdiplam (Evrysdi)
Risdiplam (Evrysdi)
Last checked: 9th July 2025
Risdiplam clinical trials and their results
Researchers have tested risdiplam in human clinical trials since 2015. Risdiplam is not a cure for 5q SMA but in these trials, it has been consistently shown to have an acceptable safety profile and to be well-tolerated. When compared to no treatment, it has led to clinically meaningful improvements in muscle function. Several trials of risdiplam are still ongoing.
Risdiplam works best if started before there is any muscle weakness, or when this is minimal. It is therefore important for treatment to be started as soon as possible.
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