Neuromuscular Clinic

Why CHKD?

CHKD's Center for Novel Therapeutics and Gene Therapy 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.

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