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Gene Therapy for DMD and SMA

Also known as: ELEVIDYS.

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.

Reviewed by: Migvis Monduy, MD

This page was last updated on: 7/6/2026 8:56:34 PM

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Related Press Releases

Nicklaus Children's Expands Offering of Approved Gene Therapy for Duchenne Muscular Dystrophy Following FDA Decision

September 18, 2024 –

Nicklaus Children’s Hospital is offering FDA-approved Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy developed for patients with Duchenne Muscular Dystrophy (DMD) who have a confirmed mutation in the DMD gene. 

patient Jacob laughs with Dr. Monduy.

Nicklaus Children's Hospital Now Administering the First FDA-Approved Gene Therapy for Duchenne Muscular Dystrophy

November 01, 2023 –

Nicklaus Children's Hospital announced it has treated its first patient with ELEVIDYS, the first gene therapy for Duchenne muscular dystrophy. Developed by Sarepta Therapeutics, ELEVIDYS is approved by the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne muscular dystrophy (DMD) in ambulatory pediatric patients ages 4 through 5 years with a confirmed mutation in the DMD gene.

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Inspiring Patient Stories

Ka'lee running down the hall holding his toy and ipad. Ka'lee is Running, Walking and Thriving After Gene Therapy for Muscular Dystrophy

Ka'lee was diagnosed with Duchenne Muscular Dystrophy (DMD) at just two years old. One of four siblings, he is the only one in his family with this condition. His mother, Carmen, did not expect the diagnosis, and at first, didn't fully understand what it would mean for her son's future. Since his first treatment in 2024 he has achieved milestones that once felt out of reach.

Learn more about

Spinal Muscular Atrophy (SMA) Spinal muscular atrophy is a rare degenerative disorder in which the nerve cells in the upper and lower parts of the spinal cord don’t function normally, resulting in muscle wasting and weakness.
Genetic Diseases Genetic diseases are conditions that occur due to a mutation in a gene in the body’s cells.
Duchenne Muscular Dystrophy Muscular dystrophy is a genetic condition characterized by progressive muscle weakness, difficulties with movement and other symptoms. Duchenne muscular dystrophy is known for getting worse much more.
Steroid Injections Steroid injections are a treatment that can be used to reduce pain and improve mobility. Steroids are injected directly into an area of the body in order to reduce pain and swelling.