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Page last checked: 14th December 2022
Last updated: 21st February 2022


Announced in October 2015, this new open-label, extension study was open to infants and children who had previously participated in the ENDEAR and CHERISH phase III clinical trials.

All people involved are aware of the drug being given, rather than the process being blinded in order to compare the drug to a placebo treatment. It is an extension study because it aims to continue the treatment of patients who have completed dosing in these previous trials.

Patients enrolled in the SHINE study will be given a 12 mg dose of ISIS-SMNRx every four months if they were previously in the ENDEAR study and every six months if they completed the CHERISH study.

This trial is due to complete in August 2023.