MDC Announce Recipients of 2024 Neuromuscular Clinical and Translational Research Grants
Muscular Dystrophy Canada (MDC) recently announced the recipients of its annual Neuromuscular Research Grants competition. Through these grants, MDC is investing $900,000 into clinical and translational research projects focused on managing healthcare, understanding diagnosis and disease progression, enhancing care, discovering novel treatments and therapies and moving research towards the development of cures.
This year, the funded studies focus on Spinal muscular atrophy, Duchenne muscular dystrophy, myotonic dystrophy, dysferlinopathies, limb-girdle muscular dystrophies, immune-mediated myopathies, autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) and cross-cutting research with findings that will inform a wide range of neuromuscular disorders. Additionally, thanks to the generosity of their supporters, MDC was able to invest $63,630 towards an innovative natural history study led by NMD4C investigator Dr. Hernan Gonorazky on Charcot-Marie-Tooth disease, bringing the organization’s investment in research projects close to $1 million.
Congratulations to NMD4C investigators Dr. Rashmi Kothary and Dr. Gerald Pfeffer, whose respective projects “Maternal transfer of AAV vectors: a minimally invasive approach to deliver SMN-gene therapy for SMA” and “Single nucleus RNAseq biomarkers in adult-onset muscle diseases” are among those that received MDC funding.
Read the press release from MDC for the full list of funded projects!
Read next...
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.