DoW Duchenne Muscular Dystrophy, Idea Development Award

The fiscal year 2026 Duchenne Muscular Dystrophy Research Program (DMDRP) Idea Development Award (IDA) promotes new ideas that are still in the early stages of development and have the potential to yield impactful data and new avenues of investigation. This award supports impactful, high-risk/high-reward research that could lead to critical discoveries or major advancements that will accelerate progress in improving outcomes for individuals with Duchenne muscular dystrophy (DMD) in the near term. Applications should include a well-formulated, testable hypothesis based on strong scientific rationale. The DMDRP strongly encourages research projects investigating therapies designed to demonstrate efficacy across the life span, including infants, toddlers, and nonambulatory individuals. Key areas of focus include: • Research that will inform and improve therapy in individuals ineligible for currently approved therapies or in-development therapeutics. • Optimizing non-adeno-associated virus (AAV) or non-viral delivery to specific tissues or cell types. • Therapies that address muscle regeneration deficits. • Strategies to overcome preexisting immunity and to facilitate repeat administration of biologic therapies. • Repopulation of the muscle compartment using cell-based therapies. • Non-genomic therapies that are downstream from the genetic defect to address disease pathogenesis. • Preclinical testing of therapies in combination with standard of care treatments or approved products. • Discovery and validation of novel targets, including genetic modifiers and factors that determine the selective vulnerability/resistance of individual muscles. • Validation of novel models to address human pathophysiology in DMD beyond skeletal muscle. • Identification of novel, minimally invasive biomarkers that allow evaluation of therapeutic effectiveness with strong potential for clinical translatability.