Duchenne muscular dystrophy (DMD) is a genetic disease characterized by the progressive deterioration of muscle fibers. It is the most common of more than 30 different types of muscular dystrophy.

There is currently no cure for DMD, but there are several treatments available that can slow the progression of the disease.

Corticosteroids

Corticosteroids such as prednisone and Emflaza (deflazacort) can increase muscle strength. However, this type of medication can cause numerous side effects, which limits their use.

Heart medications

Angiotensin-converting enzyme (ACE) inhibitors and beta-blockers can slow the course of heart muscle deterioration. Treatment with heart medications should start as soon as irregularities on an echocardiogram are detected, but before symptoms occur.

Respiratory care

When muscles used for breathing become weaker, secretions tend to accumulate in the respiratory system. A cough assist device or manually assisted coughing can help keep the respiratory system free of secretions. Eventually, assisted ventilation may become necessary to facilitate breathing.

Diet

DMD patients commonly experience constipation because of immobility and weak abdominal muscles. A diet rich in fruit and vegetables is recommended as it contains a lot of fiber and can help digestion.

Most DMD patients are not very active and take corticosteroids; these two factors favor weight gain. A balanced diet can help patients maintain a healthy weight.

Learning disabilities

About one in three patients with DMD shows signs of cognitive impairment. A neuropsychologist can prescribe educational and psychological interventions such as learning exercises and techniques.

Adaptive devices

Adaptive devices such as braces or orthoses can be used to support the ankles and feet. Regular standing improves posture and circulation, but might be challenging for DMD patients. Standing frames can make standing more comfortable.

Physiotherapy

DMD patients commonly experience contractures, a fixation of the joints that occurs in the feet, knees, hips, fingers, wrists, and elbows. Range-of-motion exercises can help prevent tendons from shortening and thereby postpone contractures.

A physiotherapist can teach the patient how to do the exercises and can recommend other activities that keep the muscles toned without causing damaging stress.

Surgery

Contractures that immobilize joints can be relieved surgically.

Many DMD patients develop scoliosis (sideward curvature of the spine) when they start using a wheelchair. A surgery that inserts metal rods with hooks into the spine can be used to straighten the spine.

Exon skipping

Exon skipping is a new molecular therapy that addresses the underlying cause of DMD. Exondis 51 (eteplirsen) is an exon-skipping treatment that could benefit about 13 percent of DMD patients. Other exon-skipping therapies are being evaluated in clinical trials.

Stop codon read-through

Stop codon read-through is another therapeutic approach addressing the underlying cause of certain types of DMD. Translarna is an investigational treatment for patients with DMD caused by a nonsense mutation. It is thought that it could enable the production of a full-length, functional dystrophin protein, which is missing in patients with DMD. Translarna has been given conditional authorization by the European Medicine Agency (EMA) but is not approved by the U.S. Food and Drug Administration.

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