Nusinersen (Spinraza)
Nusinersen (Spinraza)
Page last checked: 9th July 2025
Nusinersen clinical trials and their results
Researchers have tested nusinersen in human clinical trials since 2011. Nusinersen 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 nusinersen are still ongoing.
Nusinersen 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.
Read more about clinical trials>