Multidisciplinary management of Duchenne muscular dystrophy from childhood to adulthood

Area of Science:

• Neurology
• Genetics
• Rehabilitation Medicine

Background:

• Duchenne muscular dystrophy (DMD) is a severe, progressive neuromuscular disorder causing muscle degeneration and multisystem complications.
• Effective management necessitates a coordinated, multidisciplinary strategy focused on patient quality of life throughout their lifespan.
• Early diagnosis and proactive interventions are crucial for optimizing outcomes in DMD patients.

Purpose of the Study:

• To outline the principles of multidisciplinary care for Duchenne muscular dystrophy (DMD).
• To emphasize tailored interventions across different life stages for DMD patients.
• To address the challenges of care transition and ethical considerations in advanced DMD management.

Main Methods:

• Review of current principles in multidisciplinary care for Duchenne muscular dystrophy.
• Analysis of evolving patient needs from childhood through adulthood.
• Examination of transition programs bridging pediatric and adult care systems.
• Consideration of ethical aspects, including anticipatory directives.

Main Results:

• DMD management requires distinct, coordinated approaches for pediatric, transitional, and adult phases.
• Key interventions include physical therapy, respiratory and cardiac support, orthopedic care, and psychosocial support.
• Effective transition programs are vital for continuity of care and integration into adult life.
• Anticipatory planning and directives are essential for aligning care with patient preferences.

Conclusions:

• A lifelong, multidisciplinary approach is fundamental for improving quality of life and longevity in DMD.
• Tailored medical, psychosocial, and rehabilitative strategies are critical at each life stage.
• Addressing the complexities of care transition and ethical planning enhances patient outcomes and well-being.