Exciting News!

A groundbreaking gene therapy for Duchenne muscular dystrophy has been expanded by the FDA for children ages 4 and up. CHKD’s Chief of Neurology Dr. Crystal Proud, one of the world’s leading researchers on this treatment, is excited to offer it to more patients. Read more.

For More Information

Dr. Proud is currently accepting new neuromuscular patients as well as patients interested in Elevidys gene therapy. In June 2024, the Food and Drug Administration expanded its approval of Elevidys to include ambulatory and non-ambulatory patients who are at least 4 years old. We are happy to help coordinate logistics and travel for patients outside of our region, including international patients.

For more information, please contact  Gene.Therapy@CHKD.org, or call (757) 668-9920.

Click or tap on the link below to request more information and a consultation.

Neuromuscular Clinic - Request a Consultation Button


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 Service Line Dr. Crystal Proud.

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.

Conditions We Treat

Diagnostic Services

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.


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

Learn more about gene therapy treatment at CHKD in this video:

   

Meet Our Patients 

Jacob's Story - Spinal Muscular Atrophy



Karson's Story - Duchenne Muscular Dystrophy