Treatment Options for Muscular Dystrophy

Muscular dystrophy is a term that covers a wide range of muscle-wasting conditions, many of which are progressive and carry a variety of symptoms of differing severity.

There is no cure for any type of MD. But various medications and therapies can help manage symptoms or slow the progression of the disease. Many therapies are specific to one type of MD. Here is an overview of some common treatment options:

Medication

Patients with MD are often prescribed corticosteroids, also called glucocorticoids.

They have been shown to improve muscle strength and may slow the progression of MD. But their use can cause weight gain in the short term, brittle bones that increased the chance of fractures, cataracts, and high blood pressure over the long term.

Prednisone and Emflaza (deflazacort) are examples of corticosteroids. The U.S. Food and Drug Administration has approved Emflaza for patients with a type of MD called Duchenne muscular dystrophy (DMD).

Other medications are approved for other types of MD. For example, the FDA has approved Exondys 51 (eteplirsen) for the treatment of DMD caused by certain mutations. Translarna (ataluren) is a drug approved in Europe, but not in the U.S., to treat DMD caused by another type of mutation as well as Becker MD.

Other types of medication that MD patients commonly use include:

  • Anticonvulsants, which help control seizures and muscle spasms.
  • Immunosuppressants, which may slow damage to muscle cells.
  • Antibiotics, to treat respiratory infections.
  • Drugs to treat heart problems, such as angiotensin-converting enzyme (ACE) inhibitors and beta blockers.

Physiotherapy

Keeping active is important to strengthening muscles and improving flexibility and mobility of joints.

A physiotherapist can help design a physical therapy program tailored to each MD patient’s needs. It is important to have a specialist because too much of the wrong type of exercise can cause additional damage, depending on the type of MD the patient has.

Occupational therapy and assistive equipment

An occupational therapist can help a person with MD maintain their independence as long as possible by recommending therapy techniques, assistive equipment, and practical adaptations to maximize their quality of life. These include physical aids for all ages, such as wheelchairs, leg braces, or crutches to improve or maintain mobility.

Surgery

Doctors may recommend that some MD patients have surgery to improve or correct problems stemming from their condition.

When muscles involved in breathing start to weaken, patients may need assisted ventilation to help maintain sufficient air flow in and out of the lungs.

Initially, a patient may be given a noninvasive device, such as a mask, nosepiece, or mouthpiece. But a type of surgery called a tracheostomy may be recommended in later stages of the disease. This involves making a small opening in the neck so a tube can be inserted directly into the windpipe, or trachea. The tube can then be connected to a device to supply oxygen as necessary.

Problems swallowing caused by MD can lead to a patient getting insufficient nutrition. If this continues, doctors may recommend implanting a feeding tube that delivers nutrients directly into the stomach, through a procedure called a gastrostomy.

Other types of surgeries that an MD patient may need include:

  • Fitting a pacemaker to treat heart problems.
  • Surgery to remove cataracts.
  • Surgery to correct curvature of the spine, such as scoliosis or kyphosis.

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Muscular Dystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.