At Defeat Duchenne Canada, we know that research is the only way to find a cure and improve care for those living with Duchenne muscular dystrophy.
Since 1995, Defeat Duchenne Canada has believed in developing strong partnerships with academics and clinicians to fund the most promising research. Through their determination and love for their son, Jesse, the Davidson family built a legacy endowment fund that has granted millions of dollars to research projectsworldwide.
While science is the method behind our mission, there would be no progress without people like you.
Donations from our community, and the stories behind them, are the only reasons we have been able to fund the life-changing research milestones you will learn more about below.
To date, Defeat Duchenne Canada has funded $14.8 million across 50 research projects around the world.
Funded Projects in 2021
Dr. Niclas Bengtsson
University Of Washington – Seattle, WA
“Development of gene regulatory cassettes that enable safe- and efficient in vivo dystrophin gene-editing in muscle stem cells.”
Dr. Natasha Chang
McGill University – Montreal, QC
“Inducing stress granule formation in muscle stem cells to treat DMD.”
Dr. Ronald Cohn
The Hospital For Sick Children (SickKids) – Toronto, ON
“Simultaneous DMD gene editing and upregulation to restore protective levels of full-length dystrophin in the treatment of DMD duplication mutations.”
Dr. Sachiko Sato
Université Laval – Quebec City, QC
“Development of a Monosaccharide Therapy Using N-Acetylglucosamine to Mitigate Duchenne Muscular Dystrophy.”
Two-Year Fellowship Grant in Partnership with PPMD
Parent Project Muscular Dystrophy (PPMD), a US non-profit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), and Defeat Duchenne Canada, Canada’s leading charity fighting to defeat Duchenne, announced a collaborative research award of $172,000 (CAD) in support of a two-year Clinical Fellowship in Duchenne Endocrinology and Bone Fragility. The award will sponsor the fellowship of Dr. Kim Phung under the guidance of Dr. Leanne Ward, Professor of Pediatrics and Research Chair in Pediatric Bone Health at the University of Ottawa.
Infrastructure Grant: Satellos Bioscience
Defeat Duchenne Canada provided a research partnership and infrastructure grant to support the development of Satellos’ novel approach to treating Duchenne.
Satellos scientists are developing small molecule drugs that they believe will restore faulty regeneration and repair observed in the muscles of patients with Duchenne and potentially other degenerative muscle disorders. The company’s drug candidates regulate the activity of an enzyme, codenamed PTP-X, which Satellos discovered is involved in controlling muscle stem cell function, allowing these stem cells to properly divide and repair damaged tissue that accumulates in the muscles of Duchenne patients.
Funded Projects in 2020
Dr. Eric Hoffman
ReveraGen BioPharma – Rockville, MD
“Clinical Trial of Vamorolone in an Expanded Age Range of DMD Subjects.”
For the first time ever, Defeat Duchenne Canada has granted $1 million dollars to support a single project – the clinical trial of vamorolone in Canada. This anti-inflammatory therapy, currently under investigation, has shown promising benefits, with a better safety profile than traditional corticosteroids. Furthermore, the VBP15-006 trial is focused on collecting data in a broader age population, which remains a major gap in clinical trials today.
Dr. Dongsheng Duan
University of Missouri, Columbia – MO
“Super AAV for DMD Gene Therapy.”
Gene therapy for Duchenne is making great strides. The work undertaken by Duan and his team aims to address the delivery of these gene therapies so they are can be made affordable and available to many Duchenne patients.
Dr. Jordan Sheriko
Dalhousie University – Halifax, NS
“A survey of Canadian youth with Duchenne and Becker Muscular Dystrophy exploring gender identity, sexuality, sexual health questions and.”
Dr. Jacques P. Tremblay
Université Laval – Laval, QC
“Correction by CRISPR base editing of point mutations responsible for Duchenne Muscular Dystrophy.”
Dr. Tremblay’s laboratory aims to develop therapies to treat Duchenne individuals with point mutations affecting about 30% of Duchenne patients. His novel CRISPR approach is important as it addresses corrections to the dystrophin gene that cannot be treated by exon skipping therapies.
Dr. Lawrence Korngut
University Of Calgary – Calgary, AB
“Canadian Neuromuscular Disease Registry”
Defeat Duchenne Canada was an original funder of the CNDR, and to date, has contributed more than $1 million towards the development and maintenance of the registry.
Funded Projects in 2019
Dr. Toshifumi Yokota
University Of Alberta – Edmonton, AB
“CRISPR/Cas9-mediated Gene Editing Therapy Targeting the N-Terminal Hotspot in the DMD Gene.”
Dr. Craig Campbell
London Health Sciences Center, London ON, and Dr. Jacques Tremblay, Laval University, Quebec City, QC
“Myoblast Transplantation to Treat Boys with Duchenne.”
Dr. Michael Rudnicki
Ottawa Hospital Research Institute – Ottawa, ON
“Exosomal Delivery of Wnt7a for Treating Duchenne muscular dystrophy.”
Dr. Anthony Gramonlini
University of Toronto – Toronto, ON
“Non-viral immune modulatory nanoparticles for delivery of CRISPR/Cas9 as a treatment intervention for Duchenne Muscular Dystrophy.”
Burden of Illness Study
Muscular Dystrophy Canada (MDC), Toronto, ON
Defeat Duchenne Canada funded the study “Neuromuscular Disorders: Economic Impact and Health-Related Quality of Life” to help characterize the impact of neuromuscular disorders in Canada on affected children, adults and their caregivers, including direct costs, indirect costs, and impact on health-related quality of life.