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Duchenne is a condition that causes a person to have muscle weakness and lose muscle tissue. It is progressive, which means that it gets worse over time. Duchenne usually begins in childhood and mainly affects boys, although girls can also have it.

The main symptom of Duchenne is muscle weakness. This begins early in childhood and will continue to progress. As they get older, many people with Duchenne will require help with breathing, and may develop heart-related issues. Learn more about Duchenne symptoms.

In the past, corticosteroids have been the primary medication prescribed for Duchenne because they have been shown to slow muscle loss. However, recent research has led to new treatments for Duchenne that are designed to target the disease itself, offering renewed hope to people with Duchenne and their families. Learn more about treatment options for Duchenne.

DUVYZAT is a prescription medicine for Duchenne in patients who are 6 years of age and older. DUVYZAT was approved by the US Food and Drug Administration on March 21, 2024. DUVYZAT works differently than other treatments for Duchenne.

DUVYZAT is not a steroid. As a histone deacetylase [HDAC] inhibitor, DUVYZAT works differently than other types of Duchenne medicines to address the issues caused by lack of dystrophin. While the exact process is unknown, it is thought that HDAC inhibition helps reduce inflammation, increase muscle repair, and slow muscle loss.

DUVYZAT is for patients in the US with Duchenne who are 6 years of age and older. Duchenne is a genetic disease, which means that it's caused by changes, or mutations, in genes. Duchenne may be caused by different types of genetic mutations. Healthcare providers may prescribe DUVYZAT regardless of mutation. For more information about eligibility for treatment with DUVYZAT, please speak with your or your loved one's healthcare provider.

DUVYZAT is not limited to a specific type of genetic mutation or ability to walk, which means that patients with Duchenne 6 years of age and older may be able to take DUVYZAT. For more information about eligibility for treatment with DUVYZAT, please speak with your or your loved one's healthcare provider.

DUVYZAT was studied in a clinical trial that included 179 ambulant participants with a wide range of genetic variations of Duchenne.

DUVYZAT is indicated for the treatment of Duchenne muscular dystrophy in patients 6 years of age and older in the US. DUVYZAT is not approved for patients with Becker muscular dystrophy (BMD). We recognize the significant unmet medical needs of people living with BMD and their families. Our program for BMD is in the early stages of clinical development. It is important to note that this question pertains to an unapproved or uncleared use of DUVYZAT. For further details, please contact Medical Information at medinfo@itftherapeutics.com.

DUVYZAT is made by ITF Therapeutics, the US-based rare disease division of Italfarmaco S.p.A. Founded in 1938 in Milan, Italy, Italfarmaco is a private global pharmaceutical company that has led the successful development of many innovative therapeutic products approved for use by patients around the world. The company operates in more than 90 countries on 5 continents and continues to advance promising research to address unmet medical needs in a wide range of therapeutic areas. In January 2024, Italfarmaco launched ITF Therapeutics as a new division in the United States with a focus on the development and commercialization of products to treat rare diseases, including Duchenne. Building on a legacy grounded in collaboration and innovation, ITF Therapeutics strives to partner with leaders from the US patient advocacy and treatment communities to ensure that our programs reflect and support their unique needs and goals. The establishment of ITF Therapeutics also reflects Italfarmaco's goal to build a world-class team of experts who share a passion to make a positive impact for rare disease communities. For more information, visit ITF Therapeutics.

We are committed to working with the participants in our clinical trials to ensure they do not experience any lapse in treatment with DUVYZAT. We are coordinating with clinical trial sites to ensure proper support is provided for patients during the transition.

As a reflection of our commitment to making treatment with DUVYZAT available to as many appropriate patients in the US as possible, we will provide eligible patients with a comprehensive suite of resources through our patient services program, ITF ARC. This includes support to help patients and families navigate through insurance challenges, personalized pharmacist support, financial assistance for eligible patients, educational materials, and other resources. For more information about our patient support services, please visit the patient support page or contact ITF ARC at 1-855-4 ITF ARC (1-855-448-3272).

We understand that patients may face out-of-pocket costs in situations where insurance coverage is insufficient. As a reflection of our commitment to making treatment with DUVYZAT available to as many appropriate patients in the US as possible, we have created a suite of programs to help address potential financial concerns, including:

• A copay assistance program for commercially insured patients, where patients may pay as little as $0
• A temporary supply program that provides a limited supply of DUVYZAT to help ensure timely start for appropriate patients facing insurance-related delays
• A bridge program for patients currently on therapy who may be changing insurance providers and might otherwise experience a gap in treatment
• A patient assistance program providing DUVYZAT at no charge for eligible patients, based on established criteria