TABLE 1

Patient Outcome Goals for Each Category of Care, 2018 DMD Care Considerations

Care CategoryPatient Outcome Goal
DiagnosisAccurate, confirmed diagnosis of DMD
NeurologyComprehensive disease management that is guided by appropriate medical assessment and intervention across the whole course of the disease
RehabilitationDelay the development of contractures and deformity, minimize pain, protect skin integrity, and prolong function and ambulation
Orthopedic and/or surgical managementMaintain motor function as long as possible, minimize joint contractures, maintain a straight spine, and promote bone health
Endocrinology (bone health management)Mitigate osteoporosis progression and facilitate recovery in those with early signs of osteoporosis and in those with limited potential for medication-unassisted recovery
Endocrinology (growth and puberty)Minimize linear growth impairment, mimic normal pubertal development, and prevent life-threatening adrenal crisis
PulmonaryDecrease respiratory complications and preserve respiratory muscle function
CardiologyMaximize duration of the heart, delay the onset of heart failure and abnormalities
Gastrointestinal and nutritionPrevent undernutrition, malnutrition, and overweight or obesity
PsychosocialPsychosocial support across the life span that is used to promote thinking about the future and set expectations that individuals will actively participate in their care and daily activities
Transition of care across the life spanSuccessful navigation in the transition from adolescence to adulthood