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 worse much more.

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.

Reviewed by: Trevor J Resnick, MD

This page was last updated on: May 01, 2020 04:29 PM

A New Era of Care for Duchenne Muscular Dystrophy

November 29, 2023 – In 2023, the Food and Drug Administration (FDA) approved the first gene therapy for Duchenne muscular dystrophy (DMD). This much-needed treatment may slow or stop the progression of this debilitating and deadly disorder. Nicklaus Children’s Hospital is at the forefront of this advancement in care as one of the first hospitals in the U.S. and one of only three in Florida to offer gene therapy for DMD.