FOR PATIENTS & CAREGIVERS

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Real patient experiences with Duchenne and DUVYZAT

  • Meet Ryan

    Sixteen-year-old Ryan likes playing video games, managing his school's basketball team, and hanging out with friends. Hear what he has to share about Duchenne and DUVYZAT.

    Meet Ryan Play

    As the [basketball] manager, I am responsible to give [the team] water, fill the water bottles up, and help to support the team. I pump them up.

    — Ryan, on DUVYZAT for 4+ years

  • Meet Naythan and Ethan

    Sixteen-year-old Naythan and his 15-year-old brother, Ethan, are both living with with Duchenne. Despite the challenges that Duchenne introduces to their travel accommodations, they don’t let Duchenne hold them back. Hear how Naythan and Ethan manage the challenges of Duchenne with the help of their parents.

    Meet the Palomino family Play

    DUVYZAT has brought a hope [to] our family that we didn't have before.

    — Gisela, mother of Naythan and Ethan

  • Talking About Duchenne

    Ryan, Naythan, and Ethan open up about the ways they talk about Duchenne—turning everyday conversations with friends, family, and communities into opportunities for support, understanding, and hope.

    Talking About Duchenne Play

    Ryan Michael was diagnosed at the age of 8. My initial reaction was, what is this disease, I’ve never heard of it before…I didn't know there were different types of muscular dystrophy…

    — Susan, Ryan's mom

  • Daily Life With DUVYZAT

    Ryan, Naythan, and Ethan talk about the everyday moments that help them stay consistent with DUVYZAT—from setting reminders and organizing their day to creating routines that fit their lives. They share how small habits and support from others make a big difference.

    Daily Life With DUVYZAT Play

    I would encourage families to look online...and talk to your doctor about DUVYZAT to see if it would be something beneficial for your family.

    — Susan, Ryan's mom

After hearing from these patients, you may be wondering if DUVYZAT is right for you.
Get your questions answered at your next appointment with the DUVYZAT Discussion Guide.

Safety Information About Duchenne

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.

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.

Please see full Prescribing Information and Medication Guide.

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.

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.

Please see full Prescribing Information and Medication Guide.