Grant: Project Grant

Duchenne muscular dystrophy (DMD) is a fatal genetic disease affecting about 1 in 3,500 boys. It is caused by the absence of dystrophin, a protein that acts like a shock absorber for muscles. Without dystrophin, muscles become fragile and easily damaged, leading to progressive loss of strength. Most boys lose the ability to walk in their early teens and later develop life-threatening heart and breathing complications. Although new treatments are emerging, many are extremely expensive, have important side effects, or only work for patients with specific genetic mutations. Our research focuses on a new potential therapy based on a safe, naturally occurring sugar called N-acetylglucosamine (GlcNAc). We use a 100% pure, pharmaceutical-grade (USP) form that differs from the variable-purity supplements sold in stores. GlcNAc is found in high concentrations in human breast milk, can be taken orally, and is inexpensive to produce, making it a promising candidate for an accessible treatment for all boys with DMD. Our research suggests that GlcNAc may help in two ways: by improving the muscle's natural ability to repair and regenerate, and by reducing the chronic inflammation that damages muscle over time. We have already generated strong preliminary data showing reduced muscle damage and improved muscle function in laboratory models of DMD. This project will complete the essential preclinical studies needed to prepare for a human clinical trial in Canada. Our goal is to develop a safe, affordable therapy that could be given early in life to slow disease progression and improve quality of life for individuals and families affected by DMD.