Dr. Maryam Oskoui, McGill University, Montréal, QC

The therapeutic landscape in pediatric neuromuscular disorders has exploded in recent years. We will review the most recent advances, highlighting the level of evidence and challenges in conducting trials in rare disease. Treatment options for spinal muscular atrophy and Duchenne muscular dystrophy will be outlined.

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

  • Know the treatment options in spinal muscular atrophy;
  • Outline the therapeutic pipeline in Duchenne muscular dystrophy; and
  • Describe challenges in conducting clinical trials in rare disease.

CanMEDs Competencies addressed:
Communicator, Medical Expert, Professional, Scholar