Advancing the Future of Muscular Dystrophy Research

A Legacy Inspired by Garrett Cumming

Garrett Cumming was three years old when he was diagnosed with Duchenne muscular dystrophy (DMD), a severe genetic disorder that causes progressive muscle weakness and primarily affects boys.

Inspired by Garrett’s courage and by the experiences of other families living with muscular dystrophy, the Cumming family, friends, community members, and Muscular Dystrophy Canada came together to create a lasting investment in research. Their efforts, supported by matching funding from the Government of Alberta, established the first endowed research chair in Canada dedicated specifically to muscular dystrophy.

Established at the University of Alberta in 2011, the Chair honours Garrett’s life and legacy while supporting the search for more effective treatments for muscular dystrophy and related neuromuscular disorders. Garrett passed away in 2021, but the determination and hope that inspired this initiative continue to guide its mission.

The position is now known as the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair.

Leadership in Precision Genetic Medicine

Dr. Toshifumi Yokota was appointed as the inaugural Chair in 2011. Dr. Yokota is now a University of Alberta Distinguished Professor in the Department of Medical Genetics and leads an internationally collaborative research program focused on precision genetic medicine for neuromuscular and rare genetic diseases.

The Yokota Lab develops technologies designed to address the underlying genetic and molecular causes of disease. These approaches include:

• Antisense oligonucleotides and exon-skipping therapies

• Peptide-conjugated morpholino oligomers, including the DG9-PMO platform

• Lipid nanoparticle delivery systems

• CRISPR-based genetic medicines

• Machine-learning tools for therapeutic design

• Patient-customized and N-of-1 treatments for rare mutations

Although DMD remains a central focus, the program also investigates facioscapulohumeral muscular dystrophy (FSHD), spinal muscular atrophy (SMA), spinal and bulbar muscular atrophy (SBMA), fibrodysplasia ossificans progressiva (FOP), dysferlinopathy, and other rare genetic disorders.

From Scientific Discovery to Patient Impact

Early exon-skipping research led by Dr. Yokota and collaborators helped establish the scientific foundation for the development of viltolarsen, an antisense therapy designed to restore the dystrophin reading frame. Viltolarsen, marketed as Viltepso, received FDA approval in 2020 for patients with DMD whose mutations are amenable to exon 53 skipping.

The lab continues to address major limitations of current genetic medicines, particularly the challenge of delivering sufficient amounts of treatment to skeletal muscle, neuron, the heart, and other affected tissues.

In 2025, the lab reported that conjugating a morpholino oligonucleotide to the DG9 cell-penetrating peptide substantially improved cellular and nuclear uptake, dystrophin restoration, and skeletal- and cardiac-muscle function in a DMD mouse model. This work supports the development of more effective next-generation exon-skipping therapies.

Major Advances in 2026

In 2026, the Yokota Lab published a comprehensive characterization of DMD-Null mice, which lack all dystrophin isoforms. The study showed severe muscle weakness, reduced exercise capacity, impaired regeneration, and deficiencies in muscle stem-cell proliferation and differentiation. These findings provide new insight into the functions of shorter dystrophin isoforms and establish an important model for studying DMD biology and potential treatments.

The lab and its collaborators also demonstrated that lipid nanoparticles can improve the delivery of DUX4-targeting antisense gapmers in a model of FSHD. Repeated treatment reduced DUX4 activity, improved muscle performance, and decreased disease-related pathology, providing proof of concept for an RNA-targeted therapeutic strategy for FSHD.

Additional work is advancing patient-customized antisense treatments, artificial-intelligence-assisted oligonucleotide design, therapies targeting cardiac and skeletal muscle, and delivery platforms intended to reach tissues that remain difficult to treat.

Building the Next Generation of Researchers

The Chair supports not only scientific discovery but also the education and mentorship of undergraduate students, graduate students, postdoctoral fellows, research staff, and visiting scientists.

Trainees in the Yokota Lab gain experience across molecular biology, muscle physiology, therapeutic design, drug delivery, bioinformatics, artificial intelligence, and translational medicine. Through collaborations with academic institutions, patient organizations, biotechnology companies, clinicians, and regulatory experts, they learn how fundamental discoveries can be developed into potential treatments.

Continuing Garrett’s Legacy

The progress made through the Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair is possible because of the vision and generosity of the Cumming family, Muscular Dystrophy Canada, donors, community members, research partners, and patient families.

Their support enables bold and collaborative research that may otherwise be difficult to pursue. It allows the Yokota Lab to investigate emerging scientific questions, develop new therapeutic platforms, train future researchers, and move promising discoveries closer to patients.

Garrett’s legacy continues through every experiment, collaboration, trainee, and step toward a future in which muscular dystrophy can be treated more effectively.

Discover Our Latest Progress

The 2026 Annual Report is now available below.