Myopathies

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Treatment

Treatment depends on the cause and goals are to slow progression of the disease and relieve symptoms. Treatments range from drug therapy for the muscular dystrophies and inflammatory myopathies to simply avoiding situations that work the muscles too hard for the metabolic myopathies. Some physicians recommend that patients keep their weight down (a lighter body demands less work from the muscles) and avoid overexerting their muscles.

An incentive spirometer might improve breathing function when breathing problems develop. It is not clear that this works for many patients. Unfortunately, there is no way to strengthen the breathing muscles.

Muscular Dystrophies
The goals of treatment are to slow progression of disease and relieve symptoms. Duchenne MD and Becker MD are the subjects of current medical research and clinical trials may be available for patients with either disease.

The corticosteroids deflazacort and prednisone seem to be the most effective medications. Both improve strength and walking ability for about 6 months in boys with Duchenne dystrophy. Following initial improvement, further progression of the disease may be delayed for 3 to 5 years.

Prolonged use of corticosteroids can cause severe side effects including the following:

• Bone loss (osteoporosis)
• Depression
• High blood pressure (hypertension)
• Thinning of the skin
• Weight gain

Calcium supplements and antidepressants may be prescribed to counteract the side effects.

Preventive treatment for permanent contraction of a muscle (contractures) includes physical therapy and bracing. There are currently no drugs available to prevent or treat contractures.

Heel cord surgery (also called tendon release) and spine-straightening surgery (i.e., rod insertion) may be necessary in cases of severe contractures. Heel cord surgery is performed when the patient is still able to walk. Braces are usually required following surgery.

Endocrine Myopathies
Often, treating the underlying condition helps relieve muscle weakness and pain associated with the endocrine myopathies.

Inflammatory Myopathies
The inflammatory myopathies, such as polymyositis and dermatomyositis, are usually treated with drugs that suppress the action of the immune system. Prednisone is most commonly used to treat inflammatory myopathies. It is used initially in high doses (up to 100mg/day) and then slowly tapered to the lowest possible dose that relieves symptoms. Long-term use of prednisone can cause severe side effects .

Metabolic Myopathies
The primary goal in treating metabolic myopathies is simply to avoid situations, like strenuous exercise, that tax the muscles and promote muscle pain and weakness.

Follow-Up

People with myopathies generally visit their physician once a year, though they may be asked to make more frequent visits, depending on how the disease progresses.

Physical, Occupational, & Respiratory Therapy—The Muscular Dystrophy Association (MDA) helps patients find health practitioners certified in these therapies. Although physical therapy cannot restore already weakened muscles, it can prevent healthy muscles from weakening. Occupational and respiratory therapy help patients learn to use special equipment that can improve a person's quality of life.

MDA Social Services—The MDA provides resources that help patients and their families with financial concerns.

Genetic Counseling —Genetic counselors can provide information on the risk of passing the disease to your children.

Support Groups—Support groups help patients learn how to cope with the stress and complex range of emotions that result from chronic illness.

Flu Shot —People with neuromuscular diseases are susceptible to complications from influenza and should ask their physicians about receiving yearly flu shots.

Prognosis

If the underlying cause of the disorder can be treated successfully, as in the case of endocrine myopathies, the prognosis is usually good. Progressive myopathies that develop later in life usually have a better prognosis than conditions that develop during childhood.

Patients with Duchenne MD rarely live beyond their middle to late 20s. Patients with Becker MD may live until middle age.

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