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DMD Medications: What Are the Options?


Taking medicine can help protect the muscles and ease symptoms of DMD, even though they cannot cure the disease.


Corticosteroids the most-prescribed DMD treatment. Both the American Academy of Neurology (AAN) and the U.S. Centers for Disease Control and Prevention (CDC) guidelines recommend a special kind of steroid (called corticosteroid) for the treatment of DMD. By relieving inflammation, steroids can help stop damage and maintain muscle strength for as long as possible. And steroids can help people with DMD even after their muscles become too weak to let them walk. Corticosteroids may keep the upper limbs, heart, and lungs strong for longer, as well as reduce the chance of developing scoliosis. Your child’s treatment team can tell you more about how steroids can help slow the progress of DMD, and whether they may be appropriate for your child. To explore a DMD treatment option, please click here.

Disease-modifying drugs

By targeting the dystrophin gene, disease modifying drugs allow the body to make a version of the muscle- protecting protein which is missing in DMD. While the protein the medicine helps make can’t cure DMD, it can slow down the progression of the disease.
Disease-modifying drugs are suitable for a relatively small number of people with DMD since they only target some of the mutations that cause DMD. The only way to know for sure whether a person with DMD could benefit from a disease-modifying drug is with genetic testing.

Exon skipping

Exon skipping is a way to help the body make a form of dystrophin—the protein that people with DMD do not make naturally. By “teaching” the body to make a short version of dystrophin, the point of exon-skipping is to help the body make dystrophin again.
This kind of therapy is only available to people with a certain kind of genetic mutation, which has to be confirmed with a test.


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Date of preparation: September 2022

Important Terms and Concepts

Basic terms and key definitions

Neuromuscular disorders

Neuromuscular disorders affect the muscles and nerves, and most of the causes are genetic. This means they are either passed down through the family or caused by changes in an individual person’s genes.

Most neuromuscular disorders cause muscle weakness that worsens over time. Signs and symptoms of neuromuscular diseases can vary and may be mild, moderate, or severe.

Most often, when a child has a neuromuscular disease, they don’t grow and develop as fast as other children their age. They are often slow to start lifting their head, sitting, walking, and talking.

Treatment and supportive care may improve the symptoms of a neuromuscular disorder, increasing mobility and even life expectancy.

Muscular dystrophy

Muscular dystrophy is the term for a group of neuromuscular disorders that cause muscle weakness and muscle loss.

Duchenne muscular dystrophy (DMD) is a type of muscular dystrophy that causes muscle weakness that worsens over time. The progression and symptoms can vary from person to person.