Elevidys Now Available to Patients Ages 4 and Above, Regardless of Ambulatory Status

A breakthrough gene therapy treatment for Duchenne muscular dystrophy is now available to more patients. The FDA recently expanded its approval of Elevidys, a potentially life-saving treatment, to include children ages 4 and older, regardless of their ambulatory status. 

Affecting mostly boys, Duchenne muscular dystrophy is a devastating genetic illness that causes the loss or decrease of muscle mass over time and can lead to life-threatening heart and respiratory issues later in life. Elevidys is a single-dose infusion that helps protect and maintain muscle function by spurring the development of a protein similar to the protein missing in the muscle cells of children with Duchenne.

The recent FDA expansion marks a significant milestone for patients living with Duchenne who are awaiting treatment options. For clinicians, it means they now have a treatment option for the majority of boys and men living with Duchenne.

Dr. Crystal Proud, a neuromuscular neurologist at CHKD, lauded the FDA’s announcement to make Elevidys available to additional patients. Dr. Proud, CHKD’s director of neurology, led CHKD to become one of four centers in the nation selected to study and administer Elevidys. Last year, CHKD was the first children’s hospital in Virginia to administer Elevidys to a patient. Since then, CHKD has administered Elevidys to 26 children from Virginia and beyond.

“It’s hard to put into words what a difference gene therapy has made for my families and for my patients – especially in the world of Duchenne where there has been so much hope for so long,” Proud said. “We look forward to helping more children with this groundbreaking treatment.”

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.

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. For more information, contact Gene.Therapy@CHKD.org, or call (757) 668-9920 or click here to request additional information.

About CHKD

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