Today, ITF Therapeutics shared an update on its efforts to expand access to information, data, and treatment for the Duchenne community, including the publication of a new Plain Language Summary of the EPIDYS Phase 3 study of DUVYZAT. They also shared that more than 2,000 people with Duchenne in the U.S. have now been prescribed DUVYZAT and highlighted its ongoing PROVIDUS observational study. For more details, read the community letter below.
September 11, 2026
Dear Duchenne Community,
This Duchenne Awareness Month, we join the global Duchenne community in recognizing World Duchenne Awareness Day and this year’s theme, Access Changes Lives. We have heard from you that access means more than making new treatments available; it also means ensuring the Duchenne community has access to clear information, meaningful data, and resources that can help make informed decisions. That’s why, as we work to expand access to treatment, we are also continuing to generate, share, and make information about DUVYZAT more accessible to the community.
Making Research More Accessible
This week, a Plain Language Summary of the EPIDYS study was published in Future Rare Diseases. EPIDYS was the pivotal Phase 3 study of givinostat that supported the FDA approval of DUVYZAT® (givinostat) in Lancet Neurology April 2024. DUVYZAT is a histone deacetylase inhibitor indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older.
The EPIDYS study results were originally published in 2024, but we know that the scientific terminology used in medical journals can make research findings difficult to understand for the broader community. The new Plain Language Summary explains the study’s purpose, approach, and key findings in a more accessible format. We believe people living with Duchenne, their families, caregivers, and advocates should have meaningful access to information about research that affects their lives and we hope this resource will support informed treatment decisions and facilitate collaboration with health care providers.
Continuing to Learn
Our President, Matt Trudeau, has often said that for ITF Therapeutics, FDA approval is a beginning, not an end goal. With that in mind, we will continue to generate and share data about DUVYZAT as part of our role in the community. We currently have an observational study underway in the U.S. called PROVIDUS, an observational study designed to evaluate the safety, tolerability, and functional outcomes of people with Duchenne receiving DUVYZAT as part of routine clinical care, and we will share data when available. At the same time, our parent company, Italfarmaco, is also conducting studies globally to learn more about DUVYZAT. Together, these efforts reflect our commitment to continuing to learn about DUVYZAT and sharing what we learn with the community.
Expanding Access to Treatment
This month also marks another meaningful milestone: more than 2,000 people with Duchenne in the United States have now been prescribed DUVYZAT. Behind that number are individuals and families, as well as healthcare professionals, advocates, and others across the Duchenne community who continue to inform our work. For example, families told us that the ability to complete required lab monitoring at home could help make treatment with DUVYZAT more manageable. Based on this feedback and in consult with physician advisors, we introduced an in-home lab monitoring program to support patients starting on DUVYZAT. This program is available to all eligible patients who are prescribed DUVYZAT and deemed appropriate to participate by their physician. Reaching more than 2,000 patients is a milestone, but we still have more work to do to ensure all appropriate patients have access and reminds us of our responsibility to keep listening, learning, and working to address barriers to treatment. Access to information. Access to data. Access to treatment. All of these play a role in helping people living with Duchenne make informed decisions about their care and futures. As we recognize Duchenne Awareness Month, we express our deep gratitude to advocacy organizations and community members whose feedback continues to guide our efforts. We will continue to listen closely, collaborate fully and stay focused on making a meaningful difference—together.
Sincerely,
Caroline Allen
Head of Patient Advocacy
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.
DUVYZAT is a registered trademark of Italfarmaco S.p.A.