Treatment plans vary, but this is what you can expect from your child's medical team.
Doctors can't prevent muscular dystrophy or reverse its damage, but they can use a combination of medication and therapy to keep children who have it mobile for as long as possible.
Muscular dystrophy (MD) is a group of rare genetic diseases that cause muscles to weaken and atrophy. Over time, people with MD tend to lose control of their arms, legs, trunk, and face. The most common type of MD is Duchenne muscular dystrophy, which primarily affects young boys.
After a child is diagnosed with MD, he is set up with a team of experts, including a neurologist, pulmonologist, orthopedist, physical therapist, occupational therapist, and nutritionist. The experts work to treat the disease from every angle. "The child's total care, which goes on for his whole life, requires a lot of subspecialists doing what they need to do to help the child deal with each aspect of his condition," explains Darryl De Vivo, M.D., pediatric neurologist in the Pediatric Neuromuscular Disease Center at Columbia University Medical Center/New York-Presbyterian.
Doctors monitor children's muscle health to determine how well the muscles are working and what treatments are needed. They also do a complete cardiac evaluation regularly, since the heart muscles can weaken greatly over time.
Treatment varies, depending on the type of MD, the severity of the symptoms, and the patient's age. "Each child has a slightly different course for their disease," explains Valerie A. Cwik, M.D., executive vice president and chief medical and scientific officer of the Muscular Dystrophy Association in Tucson. The youngest patients may not need to start therapy right away because they're still gaining motor skills and getting stronger, Dr. Cwik says.
As the children get older, doctors adopt a multiprong approach, using a combination of medicine, physical therapy, and assistive devices such as leg splints to slow the disease's progress and maintain strength and function. Kids with Duchenne muscular dystrophy may also take immunosuppressants to delay some of the muscle damage.
The corticosteroid prednisone has been shown to slow muscle deterioration in boys with Duchenne. "It's not a cure, but it can help," Dr. Cwik says. Dr. De Vivo agrees: "If you do it in the right way, you can add three years to the time on his feet, so he's in a wheelchair at 15 instead of at 12," he says. Prednisone has serious side effects, however; long-term use can weaken bones and thus increase the risk of fractures. Prednisnone is also associated with weight gain, which can put added stress on weakened muscles.
Patients also undergo a variety of physical, respiratory, and speech therapies. They regularly practice range-of-motion exercises to avoid contractures; this is when limbs turn in and joints become fixed in place. The combination of muscle weakness and contractures can lead to scoliosis, a sideways curvature of the spine, and that in turn can interfere with the lungs' ability to pump air in and out of the body. Therapy can help patients stay on their feet longer. "If you can keep them standing and walking through puberty, then if they need to use a wheelchair fulltime, the risk of scoliosis goes down significantly," Dr. Cwik says.
Mobility aids, such as braces and splints, can also help. Children with Duchenne often wear ankle splints at night to prevent their Achilles tendons from shortening. Shortened tendons can make walking difficult. Other children relay on canes, walkers, and wheelchairs to get around. A standing walker or frame can help kids stand up for a few hours day to improve bone strength and circulation.
Surgery is another option. Some doctors will cut a child's tendons to loosen the joints and help her walk. Other children undergo surgery to correct scoliosis and ease related breathing issues; in this case, metal rods are placed in the body to hold the spine straight. Some kids at more advanced stages of muscular dystrophy get pacemakers to help their heart beat more regularly.
In addition to the preventative measures, researchers have promising new drugs in the works. (Check out mda.org or clinicaltrials.gov for clinical trials near you.) "I strongly advocate people trying to get involved in clinical trials whenever one is available to them," Dr. De Vivo suggests.
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