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DUVYZAT is an HDAC inhibitor

DUVYZAT is a type of medicine known as a histone deacetylase (HDAC) inhibitor. It is not a steroid and works differently than other types of medicines for Duchenne. DUVYZAT can be used for all genetic variants.

A deeper dive into HDAC inhibition

Watch the video below to learn more about how HDAC inhibition can help people living with Duchenne.

A deeper dive into HDAC inhibition Play
Talk to your healthcare provider about DUVYZAT, a unique, nonsteroidal medicine for Duchenne.
Clinical Trial Results
More than 1000 people with Duchenne are currently being treated with DUVYZAT.‡ ‡Data as of July 2025.
Stories of Strength

Hear from people living with Duchenne and from healthcare providers at our DUVYZAT Stories of Strength events.

HEAR THEIR STORIES

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, so having fewer can increase your risk of bleeding or bruising. Your doctor will check your blood count before you start DUVYZAT and regularly during treatment for signs of thrombocytopenia. Call your doctor right away if you notice unusual bleeding or small red or purple spots on the skin.
  • Increased levels of fat (triglycerides) in your blood. You may not have any symptoms, so your doctor will do blood tests before you start DUVYZAT and regularly during treatment to check your triglyceride levels.
  • 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 doctor.
  • If thrombocytopenia, increased triglycerides, or diarrhea cannot be managed, your doctor may change your dose or stop your treatment with DUVYZAT, if needed.

Before taking DUVYZAT, tell your doctor about all of your medical conditions, including:

  • any heart problems or medicines you take that could increase your chance for irregular heart rhythms.
  • any bleeding problems.

Tell your doctor 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 doctor.

What are the possible side effects of DUVYZAT?

  • DUVYZAT can cause serious side effects, including 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 doctor right away if you feel faint, have an irregular heartbeat, feel dizzy, or lose consciousness. See the section titled “What is the most important information I should know about DUVYZAT?” for more information about side effects.

The most common side effects (occurring in >5% of DUVYZAT-treated patients) included diarrhea, abdominal pain, low platelet levels, nausea/vomiting, high triglyceride levels, elevated temperature/fever, muscle aches, rash, joint pain, fatigue, constipation and decreased appetite.

These are not all of the possible side effects of DUVYZAT. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

What is DUVYZAT?

DUVYZAT is a prescription medicine 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 age 6.

Please see full Prescribing Information and Medication Guide.
More than 1000 people with Duchenne are currently being treated with DUVYZAT.‡ ‡Data as of July 2025.
Stories of Strength

Hear from people living with Duchenne and from healthcare providers at our DUVYZAT Stories of Strength events.

HEAR THEIR STORIES

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, so having fewer can increase your risk of bleeding or bruising. Your doctor will check your blood count before you start DUVYZAT and regularly during treatment for signs of thrombocytopenia. Call your doctor right away if you notice unusual bleeding or small red or purple spots on the skin.
  • Increased levels of fat (triglycerides) in your blood. You may not have any symptoms, so your doctor will do blood tests before you start DUVYZAT and regularly during treatment to check your triglyceride levels.
  • 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 doctor.
  • If thrombocytopenia, increased triglycerides, or diarrhea cannot be managed, your doctor may change your dose or stop your treatment with DUVYZAT, if needed.

Before taking DUVYZAT, tell your doctor about all of your medical conditions, including:

  • any heart problems or medicines you take that could increase your chance for irregular heart rhythms.
  • any bleeding problems.

Tell your doctor 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 doctor.

What are the possible side effects of DUVYZAT?

  • DUVYZAT can cause serious side effects, including 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 doctor right away if you feel faint, have an irregular heartbeat, feel dizzy, or lose consciousness. See the section titled “What is the most important information I should know about DUVYZAT?” for more information about side effects.

The most common side effects (occurring in >5% of DUVYZAT-treated patients) included diarrhea, abdominal pain, low platelet levels, nausea/vomiting, high triglyceride levels, elevated temperature/fever, muscle aches, rash, joint pain, fatigue, constipation and decreased appetite.

These are not all of the possible side effects of DUVYZAT. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Indication

What is DUVYZAT?

DUVYZAT is a prescription medicine 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 age 6.

Please see full Prescribing Information and Medication Guide.