Treatment Options

There's no cure for any form of muscular dystrophy. But treatment can help prevent or reduce problems in the joints and spine to allow people with muscular dystrophy to remain mobile as long as possible. Treatment options include medications, physical and occupational therapy, and surgical and other procedures.

Medications

Your doctor may recommend:

  • Eteplirsen (Exondys 51), the first medication to be approved by the Food and Drug Administration specifically to treat Duchenne muscular dystrophy. It was approved conditionally in 2016 and will continue to be evaluated during an additional two years of use.

    Although the medication appears safe, it's not clear how effective the drug is. It's definitely not a cure for DMD, but it may increase muscle strength in some people treated with the drug. Eteplirsen acts on specific gene variants that affect approximately one in seven people with DMD.

  • Corticosteroids, such as prednisone, which can help muscle strength and delay the progression of certain types of muscular dystrophy. But prolonged use of these types of drugs can cause weight gain and weakened bones, increasing fracture risk.
  • Heart medications, such as angiotensin-converting enzyme (ACE) inhibitors or beta blockers, if muscular dystrophy damages the heart.

Therapy

Several types of therapy and assistive devices can improve the quality and sometimes the length of life in people who have muscular dystrophy. Examples include:

  • Range-of-motion and stretching exercises. Muscular dystrophy can restrict the flexibility and mobility of joints. Limbs often draw inward and become fixed in that position. Range-of-motion exercises can help to keep joints as flexible as possible.
  • Exercise. Low-impact aerobic exercise, such as walking and swimming, can help maintain strength, mobility and general health. Some types of strengthening exercises also might be helpful. But it's important to talk to your doctor first because some types of exercise might be harmful.
  • Braces. Braces can help keep muscles and tendons stretched and flexible, slowing the progression of contractures. Braces can also aid mobility and function by providing support for weakened muscles.
  • Mobility aids. Canes, walkers and wheelchairs can help maintain mobility and independence.
  • Breathing assistance. As respiratory muscles weaken, a sleep apnea device may help improve oxygen delivery during the night. Some people with severe muscular dystrophy may need to use a machine that forces air in and out of their lungs (ventilator).

Surgery

Surgery may be needed to correct a spinal curvature that could eventually make breathing more difficult.

Preventing respiratory infections

Respiratory infections may become a problem in later stages of muscular dystrophy. So, it's important to be vaccinated for pneumonia and to keep up to date with influenza shots. Try to avoid contact with children or adults who have an obvious infection.


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