Support organizations for people with Duchenne

Although Duchenne is a rare disease, it boasts a strong network of support. The organizations listed below are committed to helping people living with Duchenne and their loved ones through research, education, advocacy, and community.

The Akari Foundation

The Akari Foundation

The Akari Foundation works to empower the Hispanic community with resources, awareness, advocacy, and education on rare diseases, with a focus on Duchenne.

Jett Foundation

Jett Foundation

Jett Foundation empowers people and families impacted by Duchenne through transformative programming, educational opportunities, and ongoing support. Offerings include family financial assistance, virtual support groups, and Camp Promise, a free, week-long camp and year-round program for kids, teens, and adults with neuromuscular disorders.

Muscular Dystrophy Association

Muscular Dystrophy Association

For over 70 years, Muscular Dystrophy Association (MDA) has led the way in accelerating research, advancing care, and advocating for the support of people living with muscular dystrophy, ALS, and related neuromuscular diseases. MDA's mission is to empower the people we serve to live longer, more independent lives.

Little Hercules Foundation

Little Hercules Foundation

Little Hercules Foundation serves the rare disease community through access advocacy and policy to ensure all rare disease patients get access to medically necessary treatments and care.

Cure Duchenne

Cure Duchenne

Cure Duchenne is committed to improving the lives of everyone affected by Duchenne through accelerating research to find the cure, improving care and empowering the Duchenne community.

Parent Project Muscular Dystrophy

Parent Project Muscular Dystrophy

A leading nonprofit organization dedicated to advancing research, raising awareness, and providing support for people affected by Duchenne. For over 30 years, PPMD has worked tirelessly to accelerate the development of treatments, improve care standards, and advocate for policies that benefit the Duchenne community. PPMD also offers educational resources and connects families with a network of support.

Team Joseph

Team Joseph

A non-profit founded by a mom whose son was diagnosed with Duchenne as a young boy. Their mission is to help fund cutting-edge research to find a cure for Duchenne and to support families affected by the disease. Team Joseph raises awareness, provides financial assistance for medical needs, and advocates for better care options for those living with Duchenne.

ITF Therapeutics does not control or endorse third-party organizations. As such, ITF Therapeutics makes no representation as to the accuracy or any other aspect of the information supplied by these organizations or contained on these websites. The information provided is meant for informational purposes only. It is not meant to replace your healthcare provider's medical advice.

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.