Gene Therapy for DMD and SMA

What is gene therapy?

Gene therapy offers the opportunity to treat a wide range of chronic and severe inherited diseases that previously had few to no treatment options. Genetic diseases are caused by changes (called “mutations”) in the body’s genes. Gene therapy seeks to address the underlying genetic cause of an inherited disease by inserting a functional version of the mutated gene inside cells. Nicklaus Children’s is one of only a few facilities in Florida to offer ELEVIDYS, an FDA-approved treatment for ambulatory patients with Duchenne muscular dystrophy.

What does this therapy involve?

ELEVIDYS is a one-time intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy by delaying or halting its progression with the delivery of a modified version of the dystrophin gene to muscle cells.

Is any special preparation needed?

The clinical team will first confirm the patient’s eligibility for the procedure. Then, blood tests will be taken to prepare for the treatment. In addition, patients will begin a treatment-related corticosteroid regimen one to seven days before treatment. After the infusion, patient will take part in weekly safety monitoring for three months or more, with a gradual reduction of the corticosteroid dose.

Who is a candidate for the procedure?

Candidates for the procedure must be ages 4 to 5 and ambulatory with a confirmed mutation in the DMD gene; it is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

What are the possible or likely side effects of ELEVIDYS?

The most common side effects that occurred in patients treated with ELEVIDYS include vomiting, nausea, increased liver function, fever and decreased platelet counts.

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Reviewed by: Migvis Monduy, MD

This page was last updated on: September 16, 2025 12:53 PM