What patients and families need to know
Neuromuscular Medicine Program
Treating neuromuscular diseases in children and teens requires a high level of compassion, care and expertise. The Neuromuscular Medicine Program at Children’s National Hospital leverages the expertise of an interdisciplinary team, cutting-edge research and innovative technologies to care for a wide range of neuromuscular conditions.
Choosing Children’s National for Neuromuscular Disease Care
At the Neuromuscular Program, we treat children from infancy to age 21 and we’re dedicated to giving our patients the highest quality of life in and out of a clinical setting. We offer support for social, educational and emotional difficulties that often accompany neuromuscular conditions. We also offer:
- Specialty programs and clinics. Our team offers comprehensive neuromuscular care tailored for your child through our numerous specialty clinics dedicated to specific conditions.
- The latest research and treatment options. Our team has been actively involved in clinical and basic science research in neuromuscular diseases, including Duchenne muscular dystrophy, Becker muscular dystrophy, limb girdle muscular dystrophy, congenital muscular dystrophy, hereditary neuropathy and spinal muscular atrophy. These studies are designed to identify new treatment and rehabilitation options for patients and improve medical research. We translate these findings into improving clinical care for your child. We are also proud to offer novel therapies to our neuromuscular patients, including SpinrazaTM and Zolgensma for spinal muscular atrophy and Exondys 51 for Duchenne Muscular Dystrophy. We also have an active immune therapy program for the treatment of children and adolescents with myasthenia gravis, Guillain Barre Syndrome, and Chronic Inflammatory Demyelinating Polyneuropathy (CIDP).
- Academic Excellence. Our nationally and internationally recognized team members are actively involved in organizations such as APTA, American Thoracic Society, Global Tracheostomy Collaborative, Metropolitan District of Columbia Thoracic Society, Child Neurology Society, CINRG, Cure CMD, World Muscle Society and American Society of Gene and Cell Therapy (ASGCT).
- Recognitions. Based upon the excellence in care, therapeutics and rehabilitation provided in our interdisciplinary neuromuscular program, Children's National has received multiple recognitions and care center designations including; Parent Project Muscular Dystrophy’s (PPMD) Certified Duchenne Care Center, Muscular Dystrophy Association Care Center, Cure SMA Care Center Network, Charcot-Marie-Tooth (CMT) Association Center of Excellence and Congenital Muscle Disease International Registry Clinic Partner.
Services We Provide
Our Neuromuscular Program offers the following services:
- Comprehensive clinical evaluations
- Genetic counseling: We provide in depth and compassionate genetic counseling and testing ranging from personalized sequencing panels tailored to your child’s symptoms to broad testing such as whole exome sequencing approaches.
We also offer treatments and diagnostic testing such as:
- Braces, splints, Angel Arms and WREX
- Leading-edge medications like Spinraza™ and Zolgensma, new therapies for spinal muscular atrophy, and Exondys 51 to treat Duchenne muscular dystrophy
- Nutrition counseling
- Physical therapy
- Positioning aids to help children sit, stand or lie down
- Psychological counseling
- Speech and swallowing therapy
- Electrodiagnostics (EMG)/nerve conduction studies
- Muscle ultrasound
- Skin and muscle biopsy
- Genetic testing (next generation sequencing and whole exome sequencing)
- Serologic testing (blood testing)
- Augmentative communications
- Surgical interventions
- Pulmonary function studies
- Echocardiogram/cardiac MRI
- Durable medical equipment
- Occupational therapy
To expedite evaluation, diagnosis and treatment, our specialists operate three clinics:
- The Neuromuscular Clinic is the gateway to our program. Specialists review medical histories and perform detailed clinical exams. Additional blood work, muscle ultrasound or MRI (magnetic resonance imaging), muscle biopsy, nerve conduction studies, electromyograms and genetic testing may follow to determine an underlying diagnosis.
- The Muscular Dystrophy Association (MDA) Care Center is a one-stop clinic featuring interdisciplinary care for neuromuscular diseases. In a single visit, children diagnosed with muscular dystrophies are attended to by experts in neurology, rehabilitation medicine, pulmonary medicine, cardiology, genetics, bone health, nutrition, physical and occupational therapy, and social work. Team members throughout our program are also engaged in ongoing research.
- The Diagnostic Clinic specializes in diagnosis and treatment of rare and unique neuromuscular disorders. This clinic benefits from close collaboration with experts from the National Institutes of Health.
Conditions We Treat
The Neuromuscular Program coordinates care for patients with the following conditions:
- Acquired myopathies (polymyositis, dermatomyositis)
- Acquired neuropathies and polyneuropathies (Guillain-Barre syndrome, chronic inflammatory demyelinating polyneuropathy)
- Charcot-Marie-Tooth disease/Hereditary myopathies
- Congenital muscular dystrophy (Collagen-VI, LAMA 2, alpha-dystroglycanopathy, SEPN 1)
- Congenital myasthenic syndromes
- Congenital myopathies (RYR 1, MTM 1, nemaline)
- Dejerine-Sottas disease
- Dystrophinopathies (Becker and Duchenne muscular dystrophy)
- Friedreich's ataxia
- Juvenile amyotrophic lateral sclerosis (ALS)
- Metabolic myopathies (carnitine palmityl transferase deficiency, McArdle's disease and phosphofructokinase disease (Tarui's disease))
- Mitochondrial myopathies
- Muscular dystrophies such as facioscapulohumeral muscular dystrophy (FSH or FSHD), Limb-Girdle and myotonic
- Myasthenia gravis
- Pompe disease
- Spinal muscular atrophy
Neuromuscular conditions are rare. Many are misdiagnosed or go unrecognized. The interdisciplinary team at Children’s National has the expertise to recognize these conditions and ensure a timely, accurate diagnosis. Our team includes:
Please call us at 202-476-6193 to schedule an appointment or for more information. If you're a new patient, please fill out our Neuromuscular New Patient Form before your visit. If you're a returning patient, please complete our Neuromuscular Follow-Up Form.
Neuromuscular Medicine Program Team