How is muscular dystrophy treated?

There is currently no cure for muscular dystrophy but there have been a lot of progress in understanding MDs and improving the life expectancy and quality of life of patients with MD. Steroids have now become standard of care in treating patients with Duchenne as they slow down the progression of disease. Long-term steroid use is associated with side effects and neuromuscular physicians consider those when deciding on when to start or stop steroids for patients with Duchenne MD. There are also gene therapy trials at different levels with the goal of slowing the progression of Duchenne MD. For the time being, treatment is aimed at preventing complications caused by:

  • Muscle weakness
  • Decreased mobility
  • Contractures
  • Scoliosis
  • Heart defects
  • Respiratory insufficiency

Physical and occupational therapies: Physical therapy, especially regular stretching, is important in helping to maintain range of motion for affected muscles and to prevent or delay development of contractures. Strengthening other muscles to compensate for weakness in affected muscles may be of benefit also, especially in earlier stages of milder MD. Regular exercise is important in maintaining good overall health, but strenuous exercise may damage muscles further. Customized wheelchairs, braces, handicap-friendly residential, or work environments all improve the patient’s independence and quality of life. Occupational therapy involves employing methods and tools to compensate for a patient's loss of strength and mobility. This may include modifications at home, dressing aids, wheelchair accessories, and communication aids.

Surgery: If a patient's contractures have become very pronounced, surgery may be used to relieve the tension by cutting the tendon of the affected muscle, then bracing it in a normal resting position while it heals. Surgery for scoliosis is often needed for patients with Duchenne or similar MDs.

Nutrition: Nutrition has not been shown to treat any conditioning MDs. Healthy diet is important in Duchenne patients who are on steroids as steroid-induced metabolic changes could compromise patient’s mobility and independence. Malnutrition in patients with swallowing difficulty or fatigue also needs to be addressed and alternative methods of feeding explored.

Cardiac care: Dysrhythmias or heart failure (cardiomyopathy) are a complication in dystrophinopathies (Duchenne and Becker), myotonic dystrophies, Emery-Dreifuss, and some limb-girdle MDs. Placement of cardiac pacemakers and/or different therapies for cardiomyopathy is sometimes needed in these diseases.

Respiratory care: When the muscles of the diaphragm and other respiratory muscles become very weak, a patient may require a ventilator device to continue breathing deeply enough. Air may also be administered through a tube or mouthpiece. It is therefore very important to maintain healthy lungs to reduce the risk of respiratory complications. Cough-assist devices might also be required to help clear respiratory secretions and prevent infections.

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