Treatment & Care

At the many Duchenne Certified Care Centers in the United States, you will find the top Duchenne experts in the field, the current standards of care guidelines, and the most promising clinical trial options available.

Approved Treatments

Explore the variety of Duchenne treatments below.

Exondys 51

On September 19, 2016, Exondys 51 was granted Accelerated Approval by the Food and Drug Administration (FDA), based on dystrophin as a surrogate endpoint, for the treatment of Duchenne. Exondys 51 was the first disease modifying treatment for Duchenne approved in the United States that can replace dystrophin, the protein patients with Duchenne are missing.

Exondys 51 treats approximately 13% of the Duchenne population who are amenable to the skipping of exon 51 on the dystrophin gene.

If you or someone you know has a mutation amenable to Exondys 51 and is interested in starting on therapy, contact Sarepta Assist for information on how to get started.

Emflaza (Deflazacort)

In February of 2017, the FDA approved Emflaza, a corticosteroid similar to Prednisone, for the treatment of Duchenne.

If you or someone you know is interested in beginning treatment of Emflaza, please speak with your physician about obtaining a prescription, or contact PTC Cares.

Vyondys 53

In 2019 Vyondys 53 was granted Accelerated Approval by the Food and Drug Administration (FDA). Vyondys 53 treats approximately 8% of the Duchenne population who are amenable to the skipping of exon 53 on the dystrophin gene.

If you or someone you know has a mutation amenable to Exondys 53 and is interested in starting on therapy, contact Sarepta Assist for information on how to get started.

Viltepso

In 2020, Viltepso was granted Accelerated Approval by the Food and Drug Administration (FDA). Viltepso treats approximately 8% of the Duchenne population who are amenable to the skipping of exon 53 on the dystrophin gene.

If you or someone you know has a mutation amenable to Viltepso and is interested in starting on therapy, contact NS Support for information on how to get started.

Amondys 45

In 2021 Amondys 45 was granted Accelerated Approval by the Food and Drug Administration (FDA). Amondys 45 treats approximately 8% of the Duchenne population who are amenable to the skipping of exon 45 on the dystrophin gene.

If you or someone you know has a mutation amenable to Amondys 45 and is interested in starting on therapy, contact Sarepta Assist for information on how to get started.

Types of Mutations

Duchenne is caused by mutations (changes) within the dystrophin gene. A gene is made up of coding regions called exons, and the areas in between exons are called introns. Dystrophin has 79 exons, which makes it one of the largest genes in the body.

Making the dystrophin protein from the gene involves several steps. One of the first steps is removing the introns and fitting the exons together, 1-79, like puzzle pieces.

If there is a missing piece within the dystrophin gene (deletion) or an extra piece (duplication), your body can have difficulty making dystrophin.

Visit Parent Project Muscular Dystrophy for further resources about Duchenne testing and mutations.

Insurance Flight Plan

Navigating insurance can be a tricky task, especially when you or your child requires specialized care and equipment. Our Health Insurance Flight Plan breaks down options that may be available to you and provides a launching ground for families as they begin to think about what plan may be best for them.

The booklet is free of charge and available to download online. Fill out the form below and your booklet will be on its way!

Request a Flight Plan

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