Disease Progression and Quality of Life in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) follows a predictable trajectory characterized by distinct phases of progression. The early ambulatory phase typically involves mild muscle weakness and delayed motor milestones, while the late ambulatory phase sees increasing difficulties with walking, climbing stairs, and maintaining balance. Transitioning to the non-ambulatory phases, patients experience profound loss of mobility, often requiring wheelchairs, with early non-ambulatory stages marked by upper limb weakness and emerging respiratory challenges. In late non-ambulatory stages, patients face severe respiratory and cardiac complications. These progressive physical limitations profoundly impact quality of life, affecting independence, social participation, and emotional well-being. Caregiver burden also increases over time. Understanding these stage-specific functional and psychosocial challenges is essential for guiding clinical management, tailoring interventions, and prioritizing supportive care to optimize outcomes and maintain quality of life throughout the disease course.