Page last checked: 14th December 2022

Last updated: 21st February 2022

This study was a Phase 2 trial of nusinersen that enrolled a small number of SMA patients that were ineligible for inclusion in the ENDEAR and CHERISH Phase 3 trials. Results were presented at The 22nd International Annual Congress of the World Muscle Society (WMS) held October 3rd-7th 2017.

Like CHERISH, EMBRACE was a randomised, double-blind, sham-procedure controlled study over 14 months designed to test the safety of injections of nusinersen into the spinal cord. Some information on effectiveness can also be determined from the study, although this was not its primary purpose.

The main findings from this presentation were as follows:

1. Nusinersen continued to be safe and well-tolerated.

2. Upon termination of the ENDEAR trial due to positive interim results, part 1 of the EMBRACE trial was ended, and all patients (20 children) were transferred to the open-label part 2 of the trial.

3. Before the initiation of EMBRACE part 2, a greater percentage of children (79%) receiving nusinersen showed attainment of key motor milestones compared to the sham procedure-treated (29%).

4. While both treatment groups required ventilator use, the nusinersen-treated children showed smaller increases over the duration of the trial than patients receiving the placebo.

5. There was no difference in weight gain between the two treatment groups.

Poster of Results >