Page last updated: 17th March 2023
Risdiplam Clinical Trials and Their Results
Risdiplam has been tested in human clinical trials since 2015. It has been consistently shown to 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, risdiplam has been shown to increase the availability of SMN protein, leading to clinically meaningful improvements in muscle function. Several trials of risdiplam are currently ongoing.