Neuromuscular Clinic
Neurology Clinical Trials
Why CHKD?
CHKD's Neuromuscular Clinic is an international leader in researching and delivering innovative therapeutics and gene therapy for neuromuscular conditions. We are committed to providing high-quality care and diagnostics, as well as the latest research and treatment options, so your child receives the best possible care.
Our comprehensive program not only focuses on physical health but also offers support for children who may be struggling socially and emotionally. Our goal is to empower patients to lead independent, successful, and fulfilling lives.
Exceptional Care
CHKD's neuromuscular team consists of nationally and internationally recognized providers who are actively involved in leading research, advocacy, and clinical education. Our Duchenne muscular dystrophy program is nationally recognized as a Muscular Dystrophy Association Care Center and is certified by Parent Project Muscular Dystrophy, which recognizes excellence in the care of patients with muscular dystrophy. Our Spinal Muscular Atrophy Center is also nationally recognized as an SMA care center by Cure SMA.
CHKD proudly offers Elevidys, a new FDA-approved gene therapy treatment for Duchenne muscular dystrophy. Patients around the world can receive this treatment at CHKD with our Medical Director for the Neuroscience Unit Dr. Crystal Proud.
Groundbreaking Treatment Options
CHKD offers the following novel therapeutics and gene therapies for spinal muscular atrophy and Duchenne muscular dystrophy. We ensure careful follow-up, safety monitoring, and evaluation following the initiation of these therapies.
Spinal Muscular Atrophy
- Risdiplam (Evysdi)
- Spinraza
- ZOLGENSMA
Duchenne Muscular Dystrophy
- Elevidys
- Becker muscular dystrophy
- Charcot Marie Tooth neuropathy
- Chronic immune mediated demyelinating polyneuropathy (CIDP)
- Congenital muscular dystrophy
- Congenital myopathy
- Congenital myotonia
- Duchenne muscular dystrophy
- Facioscapulohumeral muscular dystrophy (FSHD)
- Friedrich’s ataxia
- Hereditary spastic paraparesis
- Limb Girdle muscular dystrophy
- Myasthenia gravis
- Myotonic muscular dystrophy
- Spinal muscular atrophy
Each diagnostic workup and treatment plan is specifically tailored to your child. Because many nerve and muscle conditions are the result of DNA changes, our genetic counselor will discuss the need for genetic testing or the results of prior testing.
- Nerve conduction studies (NCS)/ electromyography (EMG)
- Bone density test
- X-rays to evaluate spine, hips, and joints
- Swallow study
- Sleep study
- DNA testing performed via saliva or blood testing
- Laboratory tests
Gene Therapy at CHKD
Video | Dr. Proud oversees a comprehensive treatment program offering Elevidys, a breakthrough therapy for patients with Duchenne muscular dystrophy. Learn more about our neuromuscular clinic and see how new groundbreaking treatments like Elevidys are bringing hope to patients like Karson at CHKD.
For More Information
Dr. Proud is currently accepting new neuromuscular patients as well as patients interested in Elevidys gene therapy. We are happy to help coordinate logistics and travel for patients outside of our region, including international patients.
Please contact Gene.Therapy@CHKD.org, call (757) 668-9920, or request more information and a consultation online at the link below.
