Pediatric Neuromuscular Disease
Care for Children with Duchenne Muscular Dystrophy and Other Neuromuscular Disorders
Children with neuromuscular diseases, such as Duchenne muscular dystrophy and spinal muscular atrophy, have varied needs that change with age and disease progression. In addition to strength and mobility problems, children can develop lung, heart, skeletal, and digestive conditions. Duke Children’s offers comprehensive care in one convenient location to treat these conditions and help maintain your child’s level of motor function as long as possible. We help children reach their full potential at home, in school, and in the community.
What Are Neuromuscular Diseases?
Neuromuscular diseases include hundreds of different disorders that affect the nervous system and cause progressive muscle weakness. They include:
- Muscular dystrophies
- Charcot-Marie-Tooth disease
- Spinal muscular atrophy
- Myasthenia gravis
- Mitochondrial disorders
Because neuromuscular diseases can affect so many bodily functions, your child’s care is personalized to his or her needs. Our team of experts includes specialists that address those needs. Overall, our team includes a pediatric neurologist, pediatric pulmonologist, pediatric cardiologist, physical and occupational therapists, a nutritionist, and a social worker. We also collaborate with pediatric-specific experts in endocrinology, orthopedic surgery, gastroenterology, and genetics.
Expert Care for Your Child
- We maintain a nationally recognized muscular dystrophy center. Duchenne muscular dystrophy is the most common and most severe form of muscular dystrophy. As a Certified Duchenne Care Center, a designation awarded by Parent Project Muscular Dystrophy, our center is recognized for providing the highest standards in treatment and subspecialty services.
- We specialize in treating children with spinal muscular atrophy, a rare, potentially life-threatening disease that leaves many children unable to walk, eat by mouth, or breathe independently. We intervene early with babies, helping them with basic life functions such as breathing and swallowing. We partner with families to align treatments with their values and goals.
- The pediatric pulmonologist who co-leads our neuromuscular program serves on the Medical Advisory Council for CureSMA, a national patient advocacy organization pursuing a cure for spinal muscular atrophy. He helps set national medical guidelines for children with SMA.
- We are actively involved in the latest SMA and muscular dystrophy research.
- As a member of the Cooperative International Neuromuscular Research Group, we work closely with doctors and scientists throughout the U.S. and around the world who are studying neuromuscular diseases.
- Your child may have access to innovative therapies through clinical trials.
NEUROMUSCULAR DISORDERS
Tests
Evaluating your child for neuromuscular disease includes a complete medical and family history and physical exam to check muscle strength. Tests are used to further diagnose neuromuscular disease.
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Identify abnormal genes and detect and measure certain proteins in the blood that are common in some neuromuscular diseases.
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Examines a small sample of muscle tissue -- about the size of a small pencil eraser -- under a microscope.
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Measures muscle activity with small sensors placed into the muscles.
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Measures the ability of nerves to send electrical impulses to the muscle, which can identify nerve damage.
NEUROMUSCULAR DISORDERS
Ongoing Care
There is currently no cure for neuromuscular diseases; treatment targets your child’s specific symptoms to slow disease progression and improve quality of life. For your convenience, your child’s care is all in one place -- on one floor -- at the Lenox Baker Children’s Hospital, which features attached parking and an entrance ramp. We also schedule your child’s appointments on the same day whenever possible, to minimize the inconvenience of multiple trips.
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Ongoing assessments look for early signs of problems common in children with neuromuscular diseases. We coordinate care with the right specialists, and tailor and adjust your child’s care for the highest possible physical, psychological, and social well-being. We often monitor children into young adulthood.
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Anti-inflammatory corticosteroid medication can slow muscle damage and weakness in Duchenne muscular dystrophy. Our team stays current with advancements in new and improved medicines for neuromuscular diseases. We carefully monitor your child and recommend the right time to start -- or stop -- medication.
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Physical and occupational therapy can improve range of motion and help maintain your child’s strength, mobility, and independence. Our therapy evaluations and treatments take place in a gym – not an exam room -- with ample room to play. High school and medical student volunteers often assist with gym play to make it more fun, and improve your child’s social interaction.
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When appropriate, we recommend and train your child to use walking braces and mobility devices -- such as walkers, wheelchairs, and scooters -- to help maintain independence. We also introduce a variety of assistive technologies and assistive communication devices (iPad apps, etc.) to keep your child engaged and connected with others.
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Our pediatric pulmonologist performs pulmonary function testing to identify weakness in the muscles that help your child breathe. Treatments may include a cough assist machine, a non-invasive breathing support device (CPAP or Bi-PAP), or a ventilator in more severe cases.
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The team’s pediatric cardiologist looks for signs of heart muscle weakness and deterioration through an electrocardiogram, heart ultrasound, and heart MRI testing and may recommend cardiac medications.
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Your doctor may recommend surgery for complications as your child grows and develops, such as problems with the bones and joints or the digestive system. If your child needs surgery, Duke Children’s will provide the highest quality care as we are one of only seven hospitals in the U.S. verified as a level I children's surgery center by the American College of Surgeons. This Level I designation recognizes our commitment to providing the safest and highest-quality surgical care to our young patients.
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We know how caring for a child with a neuromuscular disease can change the family dynamic. We are here to answer your questions and make your time at Duke Children’s as easy and comfortable as possible. Our clinic is staffed with a dedicated licensed clinical social worker.
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Our nurse care coordinator and social worker help you navigate the medical system and coordinate the variety of health services your child needs. They can help you work with insurance providers, connect with resources, and manage the details related to your child’s care. Our social worker can help your entire family adjust to a neuromuscular diagnosis, monitor your child’s behavioral and developmental health, and assist with school support regarding modifications for your child.
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Doctor visits and hospitals can be overwhelming and sometimes scary for children. Our child life team can enhance the care your child receives. They explain procedures in ways your child can understand, offer encouragement and emotional support, and use fun activities to take your child’s mind off treatment.