HHE Neurology supplement 2018 - Page 26

deemed to be motor milestone responders compared with none of those in the sham procedure group • At the end of the study, the proportion of responders assigned to nusinersen had increased to 51% yet again none of those in the sham procedure became responders • At the study end, all participants were given the opportunity to enter an open-label extension trial termed SHINE. The results for the second primary end-point, assessed at the study end, are shown in Table 3, i