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Duchenne Muscular Dystrophy

Also known as: DMD.

What is 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 progressively worse.

What causes Duchenne muscular dystrophy?

A genetic defect causes the body not to produce as much dystophin as it should. Dystrophin is a protein that helps strengthen muscles. However, it's unclear why this defect occurs. It does not appear to run in families.

What are the symptoms of Duchenne muscular dystrophy?

Progressive muscle weakness is the primary symptoms of Duchenne muscular dystrophy. This included problems with movement, falls and trouble getting up from sitting or lying down. Fatigue, learning problems and intellectual disability are other problems related to Duchenne muscular dystrophy.

What are Duchenne muscular dystrophy care options?

Certain drugs such as steroids can slow the loss of muscle strength. Exercises and physical therapy can help those with Duchenne muscular dystrophy live the best life possible.

Gene therapy can also address the underlying genetic cause of DMD 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.

Reviewed by: Trevor J Resnick, MD

This page was last updated on: 7/6/2026 7:21:50 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.

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Movement Disorders

Please see spasticity and movement disorders for further information.

Spasticity and Movement Disorders Spasticity is one form of movement disorder, that presents as increased tone or stiffness of the muscles that causes an inability to produce and control bodily movements.
Muscular Dystrophy (MD) Muscular dystrophy describes a broad group of many, chronic, progressive illnesses that cause the muscles of the body to become increasingly weak and less flexible over time.
Physical Therapy (PT) Evaluation and Treatment Physical therapy is a form of therapy that helps people rehabilitate through exercises, stretching and orthopedics in order to regain the mobility and function of their bodies.
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.
Gene Therapy for DMD and SMA

Gene therapy seeks to address the underlying genetic cause of an inherited disease by inserting a functional version of the mutated gene inside cells.