How Muscular Dystrophy Is Treated

Unfortunately, there is no known cure for muscular dystrophy (MD), but researchers are making advances that continue to improve the quality of lives of people living with the disease. Today, patients often turn to a variety of conventional and alternative medicine options to help treat disease symptoms and complications. The most beneficial ones very much depend on the type of MD one has and their individual case, but options range from steroids and other prescriptions to respiratory and speech therapy to surgery and more.

Prescriptions

If you have muscular dystrophy, you may need to take medications to treat the disease itself or some of its associated effects. The medications used for the treatment of muscular dystrophy can help slow the progression of muscle damage, but they do not cure the disease.

Medications you may need to take if you have muscular dystrophy include:

  • Exondys 51 (eteplirsen): This medication, approved by the U.S. Food and Drug Administration (FDA), is unique because it’s designed to treat the dystrophin protein deficiency caused by a specific genetic variant of Duchene muscular dystrophy. (Only about 13 percent of people with Duchene muscular dystrophy have that variant.)
  • Prednisone, Emflaza (deflazacort): Corticosteroids may be used to reduce inflammation, which can slow muscle degeneration in muscular dystrophy. Prednisone has been used for many years, and Emflaza was approved by the FDA in 2017 for use in muscular dystrophy for children and adults age 5 and older. 
  • Neurontin (gabapentin), Dilantin (phenytoin): Anticonvulsants, which are usually used to control seizures, are used in muscular dystrophy to control spasms and involuntary muscle activity. 
  • Antibiotics: With muscular dystrophy, you may be more prone to infections, particularly respiratory infections such as pneumonia. This is due to the weakness of the respiratory muscles. If you develop an infection, you may need to take an antibiotic for a short period of time.
  • Angiotensin-converting enzyme (ACE) inhibitors or beta blockers: Muscular dystrophy may cause heart muscle weakness, and if you experience cardiac effects of muscular dystrophy, you may need medication to improve your heart muscle function. 

You may have also heard of Translarna (ataluren), a medication that may increase normal dystrophin protein production in muscular dystrophy. This medication is not approved for use in the United States, though it is available in Europe and other regions of the world. Studies have not yet established its clinical benefit.

Therapies

Treatment for muscular dystrophy often includes supportive therapies that generally need to be scheduled on an ongoing basis. These specialized therapy sessions are tailored for muscular dystrophy and, specifically, the degree of weakness with the goal of maximizing function. 

Types of therapy that you may need if you have muscular dystrophy include:

  • Physical therapy: Physical therapy involves deliberate exercises and passive movements that train your muscles in an effort to maximize control and function and prevent excessive atrophy, which can occur due to lack of use. A therapist guides you through these in sessions and will likely provide you with instructions on exercises you can do at home (on your own or with the help of a loved one) to maintain regular muscle activity and prevent stiffness.
  • Occupational therapy: While physical therapy is focused on strength and coordination of muscles, occupational therapy is directed toward improving your ability to perform self-care tasks, get around your house, and successfully handle other daily activities.
  • Respiratory therapy: If you have respiratory muscle weakness, specialized breathing exercises can help make sure that you position yourself in a way that is advantageous for breathing. Exercising your respiratory muscles builds strength. Regularly measuring your respiratory function can alert your doctors in case you need to be treated with oxygen or use a mechanical device to help you breathe adequately. 
  • Speech therapy: If your muscles that control speech and swallowing become weak, you may need speech and swallow therapy to help you gain better control of your mouth and throat muscles. 

Devices

As the disease advances, you may need a supportive orthopedic device, such as a brace to support your leg, arm, or back. Often, a back brace is needed to hold your chest and back upright, which maximizes respiratory function as you breathe. Likewise, as MD progresses, you may require a cane or wheelchair.

Learning how to properly use these and other examples can help you not only maximize your mobility and comfort, but your safety. Speak with your orthopedist to learn more.

Specialist-Driven Procedures

If you have MD, you may need surgery to aid several issues that may result from the disease. It is difficult to predict whether you would need surgery for any of the complications that can arise from MD. Often, regular physical therapy, respiratory therapy, and use of orthopedic devices can delay the need for surgery or even make surgery unnecessary. 

Surgical interventions may include:

  • Spine surgery: Muscle weakness can result in curvature of your spine, often described as scoliosis if your muscles cannot adequately support your back as the MD progresses. The excessive curvature of the spine makes your back appear shorter. This can interfere with breathing and may also cause compression of your spinal cord, potentially causing pain, sensory loss, or paralysis. If the degree of curvature of your spine cannot be managed with a supportive back brace, you might need to have surgery, in which a rod is placed underneath your skin and near your spine for back support. 
  • Pacemaker placement: If your heart function becomes very weak and cannot be managed with medication, you may need a pacemaker, which stimulates muscle contraction. 
  • Tracheostomy tube: If you cannot power your respiratory muscles to inhale and exhale adequately, you may need to have a tracheostomy. This is a tube that is placed and sewn into the trachea (windpipe) so that you can get proper oxygen to your lungs, even with diminished muscle strength.

    Complementary Medicine (CAM)

    To date, few studies have tested the use of alternative medicine in the treatment of muscular dystrophy. If you're considering the use of any type of alternative medicine in the treatment of muscular dystrophy, it's important to consult your physician (or your child's pediatrician) before beginning treatment. Self-treating muscular dystrophy with alternative medicine and avoiding or delaying standard care may have serious consequences.

    Some options you may hear about include:

    Qigong

    Qigong—the ancient Chinese mind-body practice of linking breath, meditation, and movement—may improve well-being in people with muscular dystrophy, according to a 2004 study published in Disability and Rehabilitation. The study involved 28 patients with muscular dystrophy, some of whom reported improvements in mental, physical, and psychosocial well-being (in addition to a reduction in stress levels) after practicing qigong.

    Dietary Supplements

    Several small studies show that dietary supplements may benefit muscular dystrophy patients. For instance, a 2006 study from the American Journal of Clinical Nutrition found that supplementation with amino acids helped inhibit whole-body protein degradation (a hallmark of Duchenne muscular dystrophy). The study involved 26 boys with Duchenne muscular dystrophy, each of whom was treated with amino acid supplements for 10 days.

    Preliminary research also indicates that creatine (an amino acid that helps provide muscle cells with energy) may help treat muscular dystrophy as well. However, in a 2005 study of 50 boys with Duchenne muscular dystrophy, published in the Annals of Neurology, scientists found that six months of treatment with creatine supplements failed to improve muscle strength in participants.

    Green Tea

    Green tea may be of some benefit to people with Duchenne muscular dystrophy, according to preliminary research published in the American Journal of Physiology: Cell Physiology in 2006. In tests on mice in which they were fed green tea extract, researchers found that epigallocatechin gallate (an antioxidant found in green tea) may help protect against muscle wasting caused by muscular dystrophy. However, it's too soon to tell whether green tea may have the same effect on humans or in what form it could be beneficial.

    Sources:

    Dorchies OM, Wagner S, Vuadens O, Waldhauser K, Buetler TM, Kucera P, Ruegg UT. Green Tea Extract and Its Major Polyphenol (-)-Epigallocatechin Gallate Improve Muscle Function in a Mouse Model for Duchenne Muscular Dystrophy. Am J Physiol Cell Physiol. 2006 Feb;290(2):C616-25.

    Escolar DM, Buyse G, Henricson E, Leshner R, Florence J, Mayhew J, Tesi-Rocha C, Gorni K, Pasquali L, Patel KM, McCarter R, Huang J, Mayhew T, Bertorini T, Carlo J, Connolly AM, Clemens PR, Goemans N, Iannaccone ST, Igarashi M, Nevo Y, Pestronk A, Subramony SH, Vedanarayanan VV, Wessel H; CINRG Group. CINRG Randomized Controlled Trial of Creatine and Glutamine in Duchenne Muscular Dystrophy."Ann Neurol. 2005 Jul;58(1):151-5.

    Mok E, Eléouet-Da Violante C, Daubrosse C, Gottrand F, Rigal O, Fontan JE, Cuisset JM, Guilhot J, Hankard R. Oral Glutamine and Amino Acid Supplementation Inhibit Whole-Body Protein Degradation in Children With Duchenne Muscular Dystrophy. Am J Clin Nutr. 2006 Apr;83(4):823-8.

    Nabukera SK, Romitti PA, Campbell KA, Meaney FJ, Caspers KM, Mathews KD, Hockett Sherlock SM, Puzhankara S, Cunniff C, Druschel CM, Pandya S, Matthews DJ, Ciafaloni E, Starnet M. Use of Complementary and Alternative Medicine by Males With Duchenne or Becker Muscular Dystrophy. J Child Neurol. 2011 Dec 7.

    Wenneberg S, Gunnarsson LG, Ahlström G. Using a Novel Exercise Programme for Patients With Muscular Dystrophy. Part I: a Qualitative Study. Disabil Rehabil. 2004 May 20;26(10):586-94.

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