DUCHENNE MUSCULAR DYSTROPHY

Course overview

UNFORTUNATELY, DUCHENNE MUSCULAR DYSTROPHY (DMD) IS NOT ONLY ONE OF THE MOST SEVERE FORMS OF INHERITED MUSCULAR DYSTROPHIES BUT ALSO THE MOST COMMON HEREDITARY NEUROMUSCULAR DISEASE. SADLY, THERE IS NO KNOWN TREATMENT MODALITY THAT HALTS THE PROGRESSION OF THE DISEASE; AVAILABLE TREATMENT OPTIONS ARE PALLIATIVE. AFFECTED PATIENTS USUALLY DIE IN THEIR TWENTIES DUE TO RESPIRATORY MUSCLE WEAKNESS OR CARDIOMYOPATHY. CURRENT THERAPY IS CENTERED ON TREATMENT WITH GLUCOCORTICOIDS AND PHYSIOTHERAPY TO PREVENT ORTHOPEDIC COMPLICATIONS. THIS ACTIVITY ILLUSTRATES THE PATHOPHYSIOLOGY AND CURRENT MANAGEMENT OPTIONS FOR DMD AND HIGHLIGHTS THE ROLE OF THE INTERPROFESSIONAL TEAM IN CARING FOR AFFECTED PATIENTS AND FAMILIES.