Children's National Hospital has been actively involved in clinical and basic science or laboratory research and the Center for Genetic Medicine Research has led groundbreaking research in neuromuscular medicine. Our team utilizes up to date standard of care practices for various neuromuscular disorders, combined with expedited initiation of therapies, which may include:
- Spinraza™, ZOLGENSMA® and Evrysdi™ for spinal muscular atrophy; and
- Exondys 51, VYONDYS 53, AMONDYS 45™ and EMFLAZA® for Duchenne muscular dystrophy.
Additional disease specific therapies are used to treat conditions such as myasthenia gravis, congenital myasthenic syndrome, Pompe disease, Guillan Barre Syndrome/CIDP, infantile botulism and myotonic disorders.