Early in my career, I had the chance to watch and learn from Henri Termeer who was CEO of Genzyme and a role model to me and many others. Genzyme was a pioneering company that created unique and impactful medicines for people living with rare diseases. Henri often spoke about the importance of creating and delivering value with a servant’s mindset. Therefore, to deliver value, I need to understand what is important to you, the patient community. I need to get to know a broad spectrum of people affected by DMD. I need to hear you speak about your dreams, needs, and desires.

In my short time as a guest within the DMD community, I am grateful to have been warmly welcomed by people who have been willing to share their stories with me. At a World Duchenne Awareness Day event last month sponsored by the Jett Foundation, I met a young man from New Hampshire who has been touring the country in the family van, his faithful service pup in tow. He only has three more states to go before he hits his goal of visiting all 50 in the Union! I could have listened to this chatty guy for hours as he recounted stories from his incredible adventures. I also dined with a young man from Massachusetts who shared pictures of his summer trip to Canada where he came face to face with a moose. We marveled at the awe-inspiring – and slightly scary – sight of that mammoth creature so close he could almost touch it.

A common thread through the stories I heard that day, and in many other stories shared before and after, is that individuals with Duchenne are not defined by their disease. They are defined by the collection of their adventures and love and support of their family. I was reminded of this fact by Sam Safford’s impactful keynote address at the Jett Foundation event. Sam lives with DMD, but he does not let that one fact about him define his identity. He is a toastmaster, an advocate, and an activist. “Those who pity us should recognize that a person’s worth and potential is not defined by their physical or cognitive capabilities,” Sam explained. "What we need to strive for is the motivation of others leading to a change in their hearts.”

We know it’s asking a lot to hope that you will, in Sam’s words, “swing open the doors of your lives to reveal the unseen hardships, the unspoken fears and the uncelebrated victories that come with living with Duchenne.” We hope it helps to know that at ITF, we will always listen thoughtfully and engage with intention to be responsible servants that deliver value for all people affected by DMD. Ultimately, we aim to be the type of guests you feel good about welcoming into your community.

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.

C-DUV-US-0124 10/2024