PPMD is proud to partner with the Muscular Dystrophy Association (MDA), the FSHD Society, and the LGMD2L Foundation on a new, collaborative research grant focused on one of the most fundamental — and still unanswered — questions in neuromuscular disease: how human muscle regenerates, and how the process differs in Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), limb-girdle muscular dystrophy 2L (LGMD2L), and other neuromuscular diseases.

Through this joint effort, PPMD and partner organizations have awarded $213K to a two-year study, titled “Regenerating healthy human skeletal muscle at single nucleus resolution,” led by Abigail Mackey, PhD, at Copenhagen University Hospital, Bispebjerg Hospital.

Building a Human-Based Map of Muscle Regeneration

Dr. Mackey’s research will create the most detailed molecular map to date of how healthy human muscle repairs itself, using advanced single-nucleus analysis. Importantly, this “baseline” of normal muscle regeneration will then be directly compared with muscle samples from individuals living with Duchenne, FSHD, and LGMD2L.

This project grew out of conversations between researchers, clinicians, and patient organizations identifying gaps in the field and defining shared scientific priorities. Together, we recognize the need for modern, human-centered data that reflects the real biology of muscle regeneration across neuromuscular diseases.

Why This Matters for Duchenne

Human-based data is essential to understanding Duchenne. While animal models have advanced the field, they cannot fully capture the complexity of muscle repair in people. Identifying where and why regeneration fails in Duchenne is key to developing therapies that can preserve or restore muscle over time.

“For the Duchenne community, understanding how human muscle regenerates — and why that process fails in Duchenne — is critical to developing therapies that can meaningfully preserve and restore muscle function,” said Eric Camino, PhD, Vice President of Research & Clinical Innovation at PPMD. “This collaborative, human-centered research creates a shared scientific foundation that will accelerate better therapies for individuals living with Duchenne.”

As new therapies emerge, a clear picture of what “normal” human muscle regeneration looks like will be increasingly important. Dr. Mackey’s work will help lay that groundwork by defining healthy repair processes, identifying where regeneration diverges in Duchenne, and providing a shared resource to guide future drug development. While this research is early-stage and not a treatment itself, it represents an important investment in the science that future therapies will depend on.

PPMD is proud to collaborate on this effort and remains committed to advancing research that brings us closer to longer, stronger lives for everyone living with Duchenne.