Table of ContentsView AllTable of ContentsTreatment OptionsClinical Treatment QualificationsMedical DevicesComplementary TreatmentsSeeking Medical Care
Table of ContentsView All
View All
Table of Contents
Treatment Options
Clinical Treatment Qualifications
Medical Devices
Complementary Treatments
Seeking Medical Care
Duchenne muscular dystrophy(DMD) is an inherited muscle disease that causes weakness andatrophy(thinning of the muscles) throughout the body. This condition is not curable, but treatments can help slow down muscle atrophy and help alleviate some of the effects of this disease.
Therapy for DMD includes corticosteroids, gene therapy, and medications. Supportive devices can help alleviate the effects of DMD on the heart and lungs. A therapy approved in 2024, Duvzyat, has been shown to help slow down muscle damage in DMD.
This article discusses the treatment options for Duchenne muscular dystrophy, including medications, gene therapy, and supportive medical devices.
JohnnyGreig / Getty Images
Duchenne Muscular Dystrophy Treatment Options
Therapeutic interventions for treating DMD include treatments to help slow down muscle degeneration and medications to help alleviate the effects of DMD on the heart. Additionally, supportive equipment can help manage the effects of DMD on movement, breathing, swallowing, and the digestive system.
Some therapies are used to modulate gene activity in DMD—reducing the body’s production of harmful substances that damage the muscles.
Medications
Medications to treat DMD include treatments to help slow down muscle loss and therapies to manage medical complications.
Medications you might be prescribed to treat of DMD include:
Muscular Dystrophy Life Expectancy
Gene Therapy
Medications that interact with genes in the treatment of DMD include:
One type of gene therapy for DMD is an injection of a gene that directs protein production.Elevidys (delandistrogene moxeparvovec)is a gene that produces a short protein that resembles dystrophin. It is injected in one IV infusion.
Is DMD Curable?
Corticosteroids and Duvyzat help prevent muscle fiber damage, and gene therapy can improve dystrophin production. However, these treatments cannot fully prevent muscle degeneration in DMD.
How to Qualify for Clinical DMD Treatment
Several different mutations (genetic changes) are known to cause DMD. Not everybody who has DMD has the exact same genetic mutation.Several different mutations on the dystrophin gene have been identified in association with DMD.
Testing can determine which genetic mutation you have. Your test results will help determine which treatments could potentially benefit you.
Various medications and therapies are also restricted based on age, progression of muscle damage, and other treatments you take. A healthcare provider will assess these factors before recommending treatment.
Cost of TreatmentThe direct and indirect costs of treating DMD and providing needed support can be high.Adults and caregivers of children with DMD can benefit from consulting with a social worker on their healthcare team to see if they are eligible for Medicaid or the Children’s Health Insurance Program (CHIP).A social worker may also assist in accessing assistance programs.
Cost of Treatment
The direct and indirect costs of treating DMD and providing needed support can be high.Adults and caregivers of children with DMD can benefit from consulting with a social worker on their healthcare team to see if they are eligible for Medicaid or the Children’s Health Insurance Program (CHIP).A social worker may also assist in accessing assistance programs.
Supportive DMD Medical Devices
Living with DMD means that your muscles will become weaker, even when you use the currently available therapies. Medical devices may be necessary to help you maintain the most optimal mobility so that you can get around and also to prevent complications, such as muscle contractions.
Medical devices you may need include:
In addition to these devices, you may also have complications associated with breathing or absorbing and digesting the food you eat. Respiratory support may be necessary. You might also be given breathing exercises with anincentive spirometerto help you optimize the strength of your respiratory muscles.
What Are Muscular System Diseases?
Complementary Treatments for Symptom Management
No evidence confirms that supplements or any other complementary therapy improve or prevent muscle weakness in DMD.
If you are unable to eat an adequate diet due to limitations associated with chewing, swallowing, or constipation, you may need to take dietary supplements. Doing so can help you avoid malnutrition or excessive weight loss.
Additionally, meditation, controlled breathing, mindfulness, and exercise can help you manage your emotions, reduce stress, and maintain your best physical abilities as you are living with the effects of DMD.
Duchenne Muscular Dystrophy (DMD) Symptoms
Finding a DMD Treatment Care Team
It can be beneficial to work with an interdisciplinary team that includes medical doctors, physical therapists, occupational therapists, respiratory therapists, nutritionists,genetics counselors, social workers, and others.
You may also benefit from joining a support group so that you can meet with others who are also living with DMD. Being part of a community can help you understand that you are not alone.
Members could listen to you, suggest practical strategies for living with your condition, and even offer you an opportunity to give support to others who need it. Additionally, your loved ones may benefit from DMD caregiver support groups.
Summary
Treatment for DMD involves medication, gene therapy, and other interventions, such as physical therapy and occupational therapy to help you maximize your day-to-day abilities.
Gene therapy is one of the emerging treatment options for DMD, as it is used to help modify production of dystrophin to help prevent destruction of the muscles. At the current time, gene therapy can help improve symptoms of DMD and delay disease progression, but it is not a cure.
Additionally, gene therapy is not uniform, as a few different genetic changes cause DMD. Genetic testing can help guide your treatment.
Muscular Dystrophy vs. Multiple Sclerosis
8 Sources
Verywell Health uses only high-quality sources, including peer-reviewed studies, to support the facts within our articles. Read oureditorial processto learn more about how we fact-check and keep our content accurate, reliable, and trustworthy.
Food and Drug Administration.FDA approves nonsteroidal treatment for Duchenne muscular dystrophy. March 21, 2024.
D’Ambrosio ES, Mendell JR.Evolving therapeutic options for the treatment of Duchenne muscular dystrophy.Neurotherapeutics.2023;20(6):1669-1681. doi:10.1007/s13311-023-01423-y
Muscular Dystrophy Association.Medical management.
Food and Drug Administration.FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy. June 22, 2023.
Thayer S, Bell C, McDonald CM.The direct cost of managing a rare disease: assessing medical and pharmacy costs associated with Duchenne muscular dystrophy in the United States.J Manag Care Spec Pharm. 2017;23(6):633-641. doi:10.18553/jmcp.2017.23.6.633
Centers for Medicare and Medicaid Services.Children’s Health Insurance Program.
Boccanegra B, Verhaart IEC, Cappellari O, Vroom E, De Luca A.Safety issues and harmful pharmacological interactions of nutritional supplements in Duchenne muscular dystrophy: considerations for Standard of Care and emerging virus outbreaks.Pharmacol Res. 2020;158:104917. doi:10.1016/j.phrs.2020.104917
Meet Our Medical Expert Board
Share Feedback
Was this page helpful?Thanks for your feedback!What is your feedback?OtherHelpfulReport an ErrorSubmit
Was this page helpful?
Thanks for your feedback!
What is your feedback?OtherHelpfulReport an ErrorSubmit
What is your feedback?
