A blind national clinical trial of nusinersen involving 121 SMA - 1 patients found that babies receiving the drug showed significant motor function improvement when compared with babies in the trial who didn't get the drug. (sciencedaily.com)
Biotechnology firms Biogen and Ionis Pharmaceuticals cut short a phase - 3 clinical trial for the investigational drug nusinersen as it met endpoint... (cshl.edu)
Mice with only nusinersen didn't gain much weight past 6 grams. (sciencedaily.com)