NMD4C Members Presenting at Cure SMA Canada’s Winter Conference

About this Event, hosted by Cure SMA Canada

Thursday December 10 – Sunday December 13, 2020

Welcome to the Cure SMA Canada national SMA conference!

Cure SMA Canada are excited to be able to provide engaging, diverse, and informative topics for Canada’s SMA community to learn, heal and grow. Together we create an amazing cohesive community, learning from each other and making every tomorrow better than today.

Cure SMA would like to thank their sponsors, presenters and attendees.

NMD4C Presenters:

The NMD4C is excited to see many of its members present at this conference!

  • Mise à jour sur le Risdiplam. Dr. Maryam Oskoui
  • Nusinersen treatment update for adults with SMA. Dr. Erin K. O’Ferrall
  • Care for the Caregiver. Rhonda Teichrob
  • Spinraza – mise à jour pédiatrique. Dr. Nicolas Chrestian
  • Pediatric update on Spinraza. Dr. Hernan Gonorazky
  • Risdiplam update. Dr. Maryam Oskoui
  • Peripheral pathology in SMA: Implications for clinical care (What goes wrong in SMA and how to fix it – lessons learned from recent studies). Dr. Rashmi Kothary
  • Biomarkers for SMA. Dr. Robin Parks
  • À la recherche du role d’SMN dans les neurones et muscles : une stratégie pour l’identification de nouvelles cibles thérapeutiques pour l’Amyotrophie Spinale Infantile. Dr. Jocelyn Côté
  • Teens/adult transition and the importance of multidisciplinary care. Dr. Aaron Izenberg
  • The Changing Landscape of Respiratory Care for SMA. Dr. Reshma Amin

 

Click Here for the full Agenda

Register Now!

129442995_5326426520716803_4661792078481913206_n

Read next...

06 - 2026 Basic Research Summer School - EN

NMD4C Hosts 3rd Annual Basic Research Summer School at uOttawa!

From June 8 to 10, 2026, we hosted the 3rd annual NMD4C Basic Research Summer School at the University of Ottawa. The program supported 28 neuromuscular research trainees (graduate students and postdoctoral fellows) from across Canada and focused on developing practical skills, strengthening core knowledge in neuromuscular research, and providing opportunities to present and discuss their work. Through a combination of tutorials, lectures, hands-on workshops in cutting-edge wet lab techniques, flash talks, and both structured and informal networking, participants engaged with faculty, exchanged ideas, and built connections across institutions and career stages. 

New Publication from the NMD4C Clinical Trial Network Strengthens Trial Capacity in Canada

We are pleased to share a new publication from the NMD4C Clinical Trial Network led by Kerri Lynn Schellenberg, Homira Osman, Maria Masnata, Rhiannon Hicks, Corinne Kagan, Ana Stosic, Stacey Lintern, Erin Beattie, Drs. Hanns Lochmüller, Craig Campbell, and Jean K Mah. This work focuses on the implementation of a coordinated Canadian neuromuscular clinical trial network to enhance trial readiness, capacity, and access in rare disease research.

Canadian-Led Phase II Studies Advance Vamorolone Evidence in Duchenne Muscular Dystrophy

Canadian-Led Phase II Studies Advance Vamorolone Evidence in Duchenne Muscular Dystrophy

NMD4C investigators contributed to two phase II studies evaluating vamorolone in boys with Duchenne muscular dystrophy across early childhood and adolescence, demonstrating safety, favorable pharmacokinetics, and potential efficacy. Led by Dr. Jean Mah and conducted across Canadian clinical trial sites with national and industry collaborators, the findings supported Health Canada approval of vamorolone as the first treatment for DMD in Canada. Leaders from both the clinical and patient advocacy communities, including Defeat Duchenne Canada, highlighted the impact of collaboration among patients, families, clinicians, and researchers in advancing care and improving access to new therapies.