Dr. Cynthia Gagnon Receives 2024 Steinert Award, IDMC-15 Meeting to Take Place in Saguenay 

We are pleased to share that NMD4C steering committee member Dr. Cynthia Gagnon was honoured with a Steinert Clinical Sciences Award at the recent International Myotonic Dystrophy Consortium Meeting (IDMC-14) in Nijmegen, Netherlands! 

The prestigious Steinert awards are presented to individuals who display excellence in clinical and basic research on myotonic dystrophy. Based in Québec’s Saguenay-Lac-Saint-Jean region, an area with an elevated prevalence of Myotonic dystrophy (DM1) where 1:600 people are affected by the disease, Dr. Gagnon is the scientific director of the Groupe de recherche interdisciplinaire sur les maladies neuromusculaires (GRIMN) where she leads research projects and clinical trials on a variety of NMDs, including DM1. Dr. Gagnon has established an internationally renowned research program focused on documenting the natural history of NMDs through an interdisciplinary perspective to document disease progression and identify significant predictor and explanatory factors related to participation in daily activities and social roles of patients, and defining the best outcome measures to assess potential therapeutic targets. Dr. Gagnon is a key figure in growing the number of clinical trials available in Canada for DM1, facilitating trials at sites in Saguenay-Lac-Saint-Jean and collaborating with Dr. Hanns Lochmüller’s research team in Ottawa. 

 

Saguenay Prepares to Welcome International Colleagues to IDMC-15 in 2026 

Dr. Gagnon and GRIMN will host the 15th edition of the IDMC in Saguenay, QC in the fall of 2026 

We look forward to welcoming the leading international myotonic dystrophy conference to Canada, and will share further news on the meeting as details are available. 

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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.

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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.