Page last updated: 16th December 2022
Nusinersen trials and their results
Nusinersen has been tested in human clinical trials since 2011. It has been consistently shown to be have a well-understood safety profile and to be well-tolerated in several different trials across 5q SMA populations.
In many people who have SMA, when compared to placebo or no treatment, nusinersen has been shown to increase the availability of SMN protein, leading to clinically meaningful improvements in muscle function. Several trials of nusinersen are currently ongoing.