Various treatments are commonly used to manage the symptoms of Duchenne:

  • Illustration of a pill
    Steroids, for inflammation
  • Illustration of a person in pain
    Physical therapy, for orthopedic issues
  • Illustration of a heart complications
    Medical management, for heart or lung complications
However, these treatments don’t increase dystrophin.

When treating Duchenne, increasing dystrophin as soon as possible can be a positive step forward.

Currently, two different types of therapy can be used to increase dystrophin in people with Duchenne:

Ambassador Emmett sitting on the shoulders of his father..
Exon-skipping therapy

Helps a person with Duchenne bypass their genetic mutation to create a usable dystrophin protein within their own body.

With exon-skipping therapy, the muscle cells of a person with Duchenne make a blueprint for their dystrophin protein that’s 84-97% as long as the full-length dystrophin made by someone without DMD.

EXON-SKIPPING DYSTROPHIN* 84-97%

Each type of exon-skipping therapy works for a specific genetic mutation(s). A simple genetic test can help determine if there is an approved exon-skipping therapy for a person with Duchenne.

*For the most common mutations

How Exon-Skipping Therapy Works†

  • Puzzle pieces representing a complete dystrophin protein.

    Genes have sections called exons. The DMD gene, which makes our body's dystrophin protein, has 79 exons. Dystrophin is a key part of a group of proteins that our muscles need to function properly.

    Like interlocking puzzle pieces, these exons connect together in a very precise way to code a complete protein.

  • Puzzle pieces representing a dystrophin protein that has a missing exon.

    People with Duchenne have a mutation in one or more exons of their DMD gene. When exons don’t properly connect, their DMD gene makes an unusable form of dystrophin that cannot properly support their muscles.

    This mutation is like a missing puzzle piece (seen here as missing exon 42). Without exon 42, exons 41 and 43 cannot connect – their shapes don’t fit. Exon-skipping therapy for DMD "skips over" exons so that compatible exons (exons 41 and 44) can connect.

  • Puzzle pieces showing an exon being skipped.
  • Puzzle pieces representing a shortened but usable dystrophin protein.
With exon 43 skipped, the remaining puzzle pieces can now
connect together precisely to code a shortened but usable dystrophin protein.
Learn about an FDA-approved exon-skipping therapy for Duchenne
†Hypothetical example

About Gene Therapy

gene-therapy
Gene therapy

Inserts an engineered micro-dystrophin gene into a person with Duchenne to help their muscle cells produce a micro-dystrophin protein.

With gene therapy, the muscle cells of a person with Duchenne make a blueprint for their micro-dystrophin protein that’s 32–40% as long as the full-length dystrophin made by someone without DMD.

ENGINEERED MICRO-DYSTROPHIN 32-40%

Eligibility for gene therapy is determined by a patient’s doctor. Gene therapy is still being studied for its long-term safety and efficacy.

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Duchenne Stories

Watch and read inspiring stories of families living with Duchenne, and find out how they stay motivated to push through their challenges and live life normally.

Watch and read their stories