Corticosteroids for Duchenne muscular dystrophy
Last updated July 1, 2025, by Ashley Wong, PharmD
Fact-checked by PatrÃcia Silva, PhD
Corticosteroids, or steroids, play a central role in managing Duchenne muscular dystrophy (DMD), a progressive condition that causes muscle weakness and loss of function over time. These medications are widely used to help control symptoms of DMD and can slow disease progression.Â
Understanding how corticosteroids work, when they’re typically started, and how their side effects are managed can help people living with DMD and their caregivers make informed decisions about taking corticosteroids.
How corticosteroids help manage DMD
According to Duchenne care guidelines, corticosteroids are the mainstay of treatment for the disease.
As DMD medications, corticosteroids exert their effects by mimicking the actions of the natural hormone cortisol. This helps reduce inflammation in the body. In DMD, inflammation contributes to muscle damage, so reducing it may slow the progression of muscle weakness.
Although the exact mechanism of action of corticosteroids for DMD isn’t fully understood, they are thought to:
- stop or slow muscle breakdown
- stimulate muscle repair
- stabilize muscle fibers
- reduce inflammation and suppress overactive immune responses.
By addressing these underlying processes, corticosteroids help people with DMD maintain strength, mobility, and heart and lung function for longer.
Common side effects and how they’re managed
While there are many benefits of corticosteroids for DMD, these medications may cause a wide range of side effects, especially if taken long term.
Managing DMD with steroids may lead to:
- facial swelling and weight gain
- excessive hair growth
- acne
- fungal infections
- delayed puberty
- low bone density
- slowed growth
- behavior changes
- insulin resistance
- glucose insensitivity.
Management of these side effects varies. Some side effects, such as delayed puberty or glucose intolerance, may require further testing or referral to a specialist for proper management. While others, such as fungal infections, acne, and low bone density, can be managed with medications.
Your healthcare provider may recommend reducing or altering the dosage of your corticosteroid in certain situations, such as when your side effects are severe or particularly bothersome.
It is a good idea to talk to your healthcare provider if you develop side effects that are severe or won’t go away. They can help determine whether steroids are the cause, and if so, they can recommend potential management strategies or alternatives to corticosteroids for DMD.
Starting and stopping corticosteroid treatment
While there isn’t a specific age for starting treatment, experts recommend starting steroids for Duchenne muscular dystrophy before children experience substantial physical decline or weakness.
Most neurologists also recommend starting steroid treatment before the plateau phase of DMD, or when a child’s motor skills are not improving but haven’t gotten worse. This usually occurs around 4 to 5 years of age. Ultimately, your healthcare provider or your child’s doctor can help determine when to start steroids for Duchenne.
Length of treatment
In some DMD clinical trials, corticosteroids led to improvements in muscle strength at three months after starting treatment. However, the exact length of treatment with corticosteroids will vary from person to person.
Most people with DMD take them long term, if not lifelong, to optimize muscle strength and preserve heart and lung function.
Stopping treatment
Regardless of whether you or your child are taking corticosteroids for several weeks or years, it’s important not to stop taking any steroid medications suddenly on your own.
Stopping corticosteroids during DMDÂ treatment can lead to a life-threatening condition called adrenal crisis, which is caused by a severe and sudden drop in cortisol levels. Symptoms of adrenal crisis include:
- dehydration
- confusion
- dizziness
- high fever
- low blood pressure
- fast heart rate
- vomiting.
If you develop any signs or symptoms of adrenal crisis, get emergency medical help immediately.
If you want to stop taking steroids, be sure to talk with your healthcare provider first. They can help determine a plan for you to reduce your dosage slowly over time until it’s safe for you to stop the steroid entirely.
Working with your care team
Because long-term steroid use for DMD carries some risks, it’s important to ask your care team questions to help you better understand your treatment plan and goals. Examples may include:
- When should I start taking steroids?
- How long will I take steroids?
- Will I take the same dosage every day?
- What should I do if I develop side effects from the steroid?
- What should I do if I miss a dose of the steroid?
- When should I expect to see improvement in my condition?
Your healthcare provider will monitor you closely during treatment to ensure your dosage is appropriate, identify any side effects, and track the progression of your condition.
Talking to doctors about your symptoms or your child’s, DMD treatment options and goals, and the effectiveness of steroids can help you better manage living with DMD.
Together, you can decide which DMD medication is best and formulate a tailored treatment plan that is manageable, effective, and safe.
Muscular Dystrophy News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
