Research area: Genomic Medicine > Muscle Research

While some therapies have brought cautious optimism to the DMD community, significant challenges remain in delivering these treatments effectively, with some trials already halted due to serious side effects. The window for intervention is narrow, as once muscle is lost, it cannot be recovered.

Our team is working to develop treatments that help repair and preserve muscle in children with DMD. Using advanced human stem cell derived "muscle-in-a-dish" models derived directly from DMD patients, we can test promising new treatments and measure whether they improve muscle function before moving the best candidates forward into drug development. Crucially, this work is done in close partnership with DMD patient advocates, so that the research always reflects what matters most to the families living with this disease.

Purpose

The goal is straightforward, give children with DMD more time; more time walking, more time breathing independently, more time with their families. By identifying treatments that slow muscle loss and support repair, we hope to preserve strength and independence for longer, and extend the lives of children who deserve every possible chance.

Study details

The research program runs across two interconnected streams. First, the team builds sophisticated laboratory grown 3-dimentional (3D) muscle models that undergo a control injury to study how muscle damages and repairs. These models are then used to identify new treatments that support the repair process. The most promising of these are then tested in functional 3D mini-muscle models grown from the stem cells of children with DMD as a critical proof-of-concept step before progressing toward clinical application.