A new article examining data from clinical trials led by Melissa Armstrong, M.D., M.Sc., an assistant professor of neurology, shows that nusinersen treatment benefits certain patients with spinal muscular atrophy, or SMA, a genetic disease affecting the part of the nervous system that controls voluntary muscle movement. Before nusinersen, there were no treatments shown to benefit patients with SMA.
The article showed that efficacy is currently highest for treatment of infantile- and childhood-onset SMA in early and middle symptomatic phases.
Click here to read the article in the journal Neurology.