NMD4C: The Neuromuscular Disease Network for Canada

NMD4C is proud to celebrate two of our Investigators, Dr. James Dowling and Dr. Laura McAdam, who have been awarded research grants through the 2026 Defeat Duchenne Canada funding program! Their work is featured among three newly supported projects chosen for their promise to advance treatment approaches, strengthen care pathways, and improve the daily lives of people living with Duchenne muscular dystrophy.

Recognizing the Work of NMD4C Investigators

Dr. James Dowling

University of Pennsylvania – Pennsylvania, USA

Title: “Non Viral Gene Therapy for Duchenne Muscular Dystrophy”

Research Overview

There is a continued unmet need to develop new treatments for Duchenne Muscular Dystrophy. Viral vector delivered gene therapy of micro dystrophin has potential, and is now FDA approved in the United States. However, there are significant concerns about its safety, and it appears to be of limited effectiveness. One reason for its lack of strong benefit is the fact that this gene therapy provides only a miniaturized version of dystrophin that is 1/3 the size of the full length protein. Identifying an alternative gene therapy strategy that overcomes the limitations related to safety and dystrophin size would therefore be of great potential benefit for DMD. mRNA therapy, given using lipid nanoparticles (i.e. mRNA-LNP), is a safe potential option that enables delivery of a full length version of the dystrophin mRNA, which can be made into a fully functional dystrophin protein. mRNA-LNPs were the basis of the COVID vaccine, and now have been proven effective for rare genetic metabolic diseases, and have shown great benefit and safety. The also present the ability to be made at scale at a fraction of the cost of viral gene therapy.

A key barrier for mRNA-LNP therapy for muscle diseases is the challenge of delivering LNPs to muscle after ‘systemic’ (intravenous) administration. In this proposal, we will overcome this barrier, and develop and delivery a safe and effective full length dystrophin mRNA to all of the muscles in the body. For this study, we will execute on this plan in a compressed time period (2 years) using the mouse model of the disease, recognizing its limitations as a predictive model but emphasizing its strengths for enabling the testing of 100s of mRNA-LNP combinations in a short time period. The goal is to then rapidly translate our discovery to the clinic, as we have done for other mRNA therapies. Successful development of this potentially transformative therapeutic approach is aided by the experience of the two principal investigators, one (Weissman) who is a world leading pioneer of mRNA-LNP, and the other (Dowling) who is a recognized international expert in therapy development for childhood muscle diseases.

Learn More About Dr. Dowling’s Work

Dr. Laura McAdam

Holland Bloorview Kids Rehabilitation Hospital – Toronto, Canada

Title: “Youth Helping Youth: Dissemination of a user-informed bullying resource for youth with Duchenne Muscular Dystrophy across Canada”

Research Overview

Despite increasing awareness and anti-bullying strategies, bullying is a global problem that impacts children and adolescents. Children with a disability are 1.7 times more likely to be bullied than children without a disability. Despite research on bullying amongst children and youth with disabilities, there was a gap in research about the bullying experiences of children and youth with progressive muscle weakness such as Duchenne Muscular Dystrophy (DMD).

The project team conducted a research study to explore the frequency and experiences of bullying of youth with neuromuscular diseases like DMD. Findings resulted in a knowledge mobilization product, the Bullying Report, which is a 10-page document containing tangible strategies and recommendations for coping with bullying and bullying prevention for key stakeholders (youth, families, schools, clinicians) informed by youth participants.

Now it is time to disseminate the Bullying Report widely to the DMD community across Canada. The objectives of the proposed study include: 1) prepare the Bullying Report for dissemination; 2) disseminate the Bullying Report; and, 3) evaluate its reach and impact. We will share the Bullying Report in accessible formats for use across Canada and evaluate it using the RE-AIM framework. Implementation of the Bullying Report may enable youth with DMD and their families to confidently take pro-active action to minimize bullying and build confidence in navigating possible bullying events. Anticipate outcomes include a positive impact on quality of life.

Learn More About Dr. McAdam’s Work

Stronger Together in the Fight Against Duchenne

This year’s grants are part of Defeat Duchenne Canada’s long standing commitment to investing in research across the full spectrum of scientific discovery, clinical innovation, and psychosocial support. The organization recently surpassed a major milestone of more than twenty million dollars invested in Duchenne research over thirty years, demonstrating the strength and generosity of the Canadian Duchenne community. These newly funded studies led by Dr. Dowling and Dr. McAdam contribute directly to the momentum needed to advance life changing treatments and improve support systems for individuals and families across Canada. Together with the broader community of researchers, clinicians, families, and partners, their work brings us one step closer to a future where every person affected by Duchenne can live a longer and better life.

The NMD4C expresses its heartfelt congratulations to both investigators. Their dedication, expertise, and compassion reflect the very best of our national research network. We are proud to celebrate their achievements and look forward to the meaningful impact their work will continue to have on the Duchenne community.

Read More about DDC’s 2025 Research Grants