Duchenne muscular dystrophy, or DMD, is the most prevalent childhood form of muscular dystrophy, reports that National Institute of Neurological Disorders and Stroke. The aberrant gene associated with DMD leads to an abnormal form of the protein dystrophin. The defective dystrophin causes progressive muscular degeneration, weakness and loss of function. Treatments for DMD focus on slowing disease progression, optimizing existing functional capacity and preventing complications.
Glucocorticoids
Glucocorticoids are the primary medical therapy for Duchenne muscular dystrophy, according to the DMD Care Considerations Working Group commissioned by the Centers for Disease Control and Prevention. In a 2009 review article published in "The Lancet Neurology," Dr. Katharine Bushby and her working group colleagues report that glucocorticoids remain the only therapy known to forestall the progressive loss of muscle function associated with DMD.
The DMD Care Considerations Working Group notes that maximum benefits are realized if glucocorticoid therapy begins before a child's motor function begins to decline, a phase of the illness known as the plateau. Most children with DMD reach the plateau between the ages of 4 to 8 years. After completion of all childhood immunizations, Bushby and colleagues recommend starting children in the DMD plateau phase on 0.75 mg/kg of prednisone daily, with dosage adjustments as needed.
Physical Therapy and Orthopedic Braces
Patients with DMD are prone to flexion joint contractures--stiffening of the joint in a bent position with limited ability to straighten the limb. Regular physical therapy with gentle stretching and strengthening tailored to the needs of the child can prevent or reduce the severity of flexion contractures and preserve joint mobility, advises the patient information website Kids Health, sponsored by the Nemours Foundation. Similarly, orthopedic braces help prevent contractures and may prolong the time a child with DMD can continue to walk.
Tendon Release Surgery
Surgery to release the tendons associated with joint contractures of the legs may help keep a patient with DMD walking, reports the American Academy of Orthopaedic Surgeons. Among patients who are unable to walk, tendon release surgery may provide relief from pain associated with contractures.
Respiratory Therapy
Children with DMD are prone to respiratory problems and infections due to progressive weakness of the muscles involved with breathing. The DMD Care Considerations Working Group recommends respiratory therapy for children with DMD, including manual or mechanical coughing assistance to help clear lung secretions and assisted ventilation at night, as needed. With disease progression, children often require intermittent or continuous daytime ventilator assistance.
Heart Medications
The muscle cells of the heart are affected by DMD, often causing significant cardiac malfunction as the disease progresses. The DMD Care Considerations Working Group recommends early intervention with heart medications in patients with evidence of compromised cardiac function. The working group notes that angiotensin converting enzyme inhibitors, beta-blockers and diuretics are often useful for patients with heart disease associated with DMD.
References
- National Institute of Neurological Disorders and Stroke: Muscular Dystrophy
- Genetics Home Reference: DMD
- "The Lancet Neurology"; Diagnosis and Management of Duchenne Muscular Dystrophy, Part 1: Diagnosis, and Pharmacological and Psychosocial Management; Katharine Bushby, M.D., et al.; November 2009
- Kids Health from Nemours: Muscular Dystrophy, Caring for a Child with MD
