Muscular Dystrophy Canada Statement on Collaboration with Novartis for National Newborn Screening in Spinal Muscular Atrophy (SMA)
This past week, Muscular Dystrophy Canada (MDC) released a statement from Stacey Lintern, Chief Executive Officer of MDC on a Collaboration with Novartis for National Newborn Screening in Spinal Muscular Atrophy (SMA).
Statement from Dr. Hanns Lochmüller, Lead Investigator for NMD4C:
“We are thrilled to see our partners MDC collaborating with Novartis Pharmaceuticals Canada Inc to implement newborn screening as a standardized procedure across Canada. The strong national network of patient organizations, medical experts, researchers, and other stakeholders combined with investment from Novartis is a robust foundation on which to develop a national newborn screening plan.”
For many neuromuscular disorders, early diagnosis and prompt access to treatments are integral in achieving positive outcomes.
Unfortunately, this is not the reality for many patients who do not receive diagnoses until much later. For children born with Spinal Muscular Atrophy (SMA), newborn screening can identify children in need of treatment and care, which can drastically increase their chances of positive developmental outcomes.
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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
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.