Learn about Duchenne muscular dystrophy
Finding out that you or a loved one has Duchenne can be overwhelming. Whatever you are feeling—be it fear, sadness, anger, or something else entirely—it is completely normal.
Though there will be challenges ahead, learning about Duchenne can be empowering. Understanding as much as you can will help you make informed decisions about treatment options, care strategies, and ways to improve quality of life. Knowledge also allows you to advocate more effectively for yourself or your loved one, connect with support networks, and find strength in the shared experiences of others facing similar journeys.
Remember, you're not alone. Taking the time to learn about Duchenne is a crucial step in navigating this path with hope and resilience.
What is Duchenne?
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Duchenne muscular dystrophy (often called DMD or Duchenne) is a rare genetic disorder that mainly affects boys. Duchenne symptoms begin to appear in early childhood. People with Duchenne have a mutation (or change) in a specific gene that causes this disease. This leads to a lack of dystrophin and histone deacetylase (HDAC) overactivity. Dystrophin is a protein that plays a role in muscle strength, and HDAC is an enzyme that—when overactive—interferes with the body's ability to repair damaged muscle.
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When someone has Duchenne, their muscles get weaker over time. This makes it more difficult for them to walk, lift their arms, and perform daily tasks. Eventually, people with Duchenne lose the ability to walk. In addition to physical symptoms, people with Duchenne may also have intellectual differences, including difficulties with attention, memory, and learning.
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Duchenne is a progressive condition, which means it gets worse over time and cannot be reversed. For that reason, early diagnosis and treatment are important. Although there is no cure yet, treatment options are available to help those with Duchenne. Some treatments may help manage symptoms while others may slow the progression of the disease.
How is Duchenne treated?
Duchenne is typically treated through a combination of medicine, mobility assistance, and therapy. In the past, corticosteroids have been the primary medication prescribed for Duchenne because they have been shown to slow muscle loss. However, recent research has led to new treatments that are designed to target the disease itself, offering renewed hope to people with Duchenne and their families.
Strategies for supporting mobility and comfort are also important and may vary depending on a person’s age and abilities.
Leg braces and walkers can help children with Duchenne remain active as long as possible. As a child grows older, transitioning to a wheelchair may actually increase their independence. For caregivers, things like mechanical lifts, shower chairs, and electronic beds can be very helpful.
- Physical therapy to retain muscle strength
- Respiratory therapy to help with breathing issues
- Occupational therapy to help with swallowing difficulties
- Behavioral therapy to help with cognitive function
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Mobility assistance -
Therapy interventions -
Prescription treatments
Real Stories
Indication and Important Safety Information
What is DUVYZAT?
DUVYZAT is a prescription medicine that is used for the treatment of Duchenne muscular dystrophy (DMD) in people 6 years of age and older.
It is not known if DUVYZAT is safe and effective in children under 6 years of age.
Important Safety Information
What is the most important information I should know about DUVYZAT?
- Low platelet counts in your blood (thrombocytopenia). Platelets are important for blood clotting, and a decrease in their numbers can lead to an increased risk of bleeding or bruising. Your healthcare provider will check your blood count before you start DUVYZAT and regularly during treatment for any signs of thrombocytopenia. Call your healthcare provider right away if you notice any unusual bleeding or small red or purple spots on the skin called petechiae. Your healthcare provider may change your dose of DUVYZAT if your blood platelet counts continue to be low or may stop your treatment with DUVYZAT.
- Increased levels of fat (triglycerides) in your blood. You may not have any symptoms, so your healthcare provider will do blood tests before you start DUVYZAT and regularly during treatment to check your triglyceride levels. Your healthcare provider may change your dose of DUVYZAT if your triglyceride levels continue to be high or may stop your treatment with DUVYZAT.
- Frequent watery loose stools (diarrhea) and vomiting. DUVYZAT can cause vomiting and moderate to severe diarrhea. If diarrhea occurs, you should keep track of the frequency and severity of your diarrhea symptoms, drink plenty of fluids, and contact your healthcare provider. Your healthcare provider may change your dose of DUVYZAT if the diarrhea cannot be managed or does not go away. Your healthcare provider may also stop your treatment with DUVYZAT.
Before taking DUVYZAT, tell your healthcare provider about all of your medical conditions, including if you:
- have any heart problems or if you take any medicines that could increase your chance for irregular heart rhythms.
- have any bleeding problems.
Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Taking DUVYZAT with certain other medicines may affect each other. Taking DUVYZAT with other medicines can cause serious side effects. Do not start or stop other medicines without talking to your healthcare provider.
DUVYZAT can cause serious side effects, including:
- See “What is the most important information I should know about DUVYZAT?”
- changes in the electrical activity of your heart called QT Prolongation. QT Prolongation can increase the risk of developing a type of irregular heart rhythm known as Torsades de Pointes. Call your healthcare provider right away if you feel faint, have an irregular heartbeat, feel dizzy, or lose consciousness.
The most common side effects of DUVYZAT included diarrhea, nausea, vomiting, stomach pain, low platelet counts in the blood, increased fat level in the blood, and fever.
These are not all of the possible side effects of DUVYZAT. For more information, ask your healthcare provider or pharmacist.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Please see full Prescribing Information and Medication Guide.Indication and Important Safety Information
What is DUVYZAT?
DUVYZAT is a prescription medicine that is used for the treatment of Duchenne muscular dystrophy (DMD) in people 6 years of age and older.
It is not known if DUVYZAT is safe and effective in children under 6 years of age.
Important Safety Information
What is the most important information I should know about DUVYZAT?
- Low platelet counts in your blood (thrombocytopenia). Platelets are important for blood clotting, and a decrease in their numbers can lead to an increased risk of bleeding or bruising. Your healthcare provider will check your blood count before you start DUVYZAT and regularly during treatment for any signs of thrombocytopenia. Call your healthcare provider right away if you notice any unusual bleeding or small red or purple spots on the skin called petechiae. Your healthcare provider may change your dose of DUVYZAT if your blood platelet counts continue to be low or may stop your treatment with DUVYZAT.
- Increased levels of fat (triglycerides) in your blood. You may not have any symptoms, so your healthcare provider will do blood tests before you start DUVYZAT and regularly during treatment to check your triglyceride levels. Your healthcare provider may change your dose of DUVYZAT if your triglyceride levels continue to be high or may stop your treatment with DUVYZAT.
- Frequent watery loose stools (diarrhea) and vomiting. DUVYZAT can cause vomiting and moderate to severe diarrhea. If diarrhea occurs, you should keep track of the frequency and severity of your diarrhea symptoms, drink plenty of fluids, and contact your healthcare provider. Your healthcare provider may change your dose of DUVYZAT if the diarrhea cannot be managed or does not go away. Your healthcare provider may also stop your treatment with DUVYZAT.
Before taking DUVYZAT, tell your healthcare provider about all of your medical conditions, including if you:
- have any heart problems or if you take any medicines that could increase your chance for irregular heart rhythms.
- have any bleeding problems.
Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
Taking DUVYZAT with certain other medicines may affect each other. Taking DUVYZAT with other medicines can cause serious side effects. Do not start or stop other medicines without talking to your healthcare provider.
DUVYZAT can cause serious side effects, including:
- See “What is the most important information I should know about DUVYZAT?”
- changes in the electrical activity of your heart called QT Prolongation. QT Prolongation can increase the risk of developing a type of irregular heart rhythm known as Torsades de Pointes. Call your healthcare provider right away if you feel faint, have an irregular heartbeat, feel dizzy, or lose consciousness.
The most common side effects of DUVYZAT included diarrhea, nausea, vomiting, stomach pain, low platelet counts in the blood, increased fat level in the blood, and fever.
These are not all of the possible side effects of DUVYZAT. For more information, ask your healthcare provider or pharmacist.
Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Please see full Prescribing Information and Medication Guide.You are now leaving this site
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