Through expert presentations, participants will learn about exciting advances in gene therapy for the treatment of neuromuscular diseases, including SMA.  Speakers will also explore the critical role newborn screening plays in facilitating pre-symptomatic diagnosis and early treatment of disease, and the implementation of newborn screening in Canada. The session will be followed by an interactive live Question & Answer session with all speakers.

Learning Objectives:

Upon completion of this program, participants will be better able to:

  • Discuss the current clinical research evidence and application of gene therapy in paediatric neurological and neuromuscular diseases (NMD)
  • Appraise the role of current treatments in helping SMA patients reach their milestones
  • Summarize the key considerations, learnings and outcomes related to public and private sectors working on newborn screening (NBS)
  • Determine the role of the clinical and research community in facilitating the inclusion of additional NMDs’ into NBS


Jim Dowling MD, PhD, FRCPC

Senior Scientist, Genetics & Genome Biology, SickKids Hospital

Toronto, ON

Hugh McMillan MD, MSc, FRCPC, FAAN

Pediatric Neurologist, McGill University Health Centre

Montreal, QC

Pranesh Chakraborty MD, FRCPC, FCCMG

Paediatrician, The Children’s Hospital of Eastern Ontario

Ottawa, ON

Stacey Lintern

Chief Executive Officer, Muscular Dystrophy Canada

Toronto, ON

Homira Osman PhD

VP of Research & Public Policy at Muscular Dystrophy Canada

Toronto, ON

This learning activity was made possible with support from Novartis Pharmaceuticals Canada Inc.