Dr. Craig Campbell, Children’s Hospital London Health Sciences Centre, London, ON

There are now multiple novel therapies for SMA that have been shown effective in clinical trials, with nusinersen being the first approved therapy in Canada. The pathogenetic process of SMA will be reviewed and new therapy mechanisms will be presented. Data from clinical trials with a focus on respiratory outcomes will be presented. The focus will be on pediatric patients and trials.

Learning Objectives
At the end of this presentation, attendees will be able to:

  • Understand the clinical presentation and underlying genetic underpinning for SMA;
  • Describe the new therapeutic options for children with SMA; and
  • Determine the best outcome measures in research and clinical practice for SMA with a focus on respiratory impact.