Nusinersen Clinical Trials and Their Results
Nusinersen Clinical Trials and Their Results
Page last checked: 29th November 2022
Last updated: 21st February 2022
Nusinersen was the first drug developed specifically to treat 5q SMA.
The ENDEAR trial was ended early in August 2016 when the interim analysis of what were then 20 children showed positive results that would have made it unethical for the control group of children to continue with a sham procedure. Biogen then went on to open its global Expanded Access Programme (EAP) for SMA Type 1.
Results from the CHERISH trial and other studies around this time were key to nusinersen being approved for use in many countries.
Trials of nusinersen continue to assess its clinical efficacy for children who are presymptomatic. Other trials are assessing higher doses and other possibilities