Duchenne Drug Development Pipeline

The Drug Development Pipeline is full of potential treatments that are being tested. These include therapeutic approaches that restore or replace dystrophin and those that treat Duchenne symptoms (such as those that protect muscles by reducing fibrosis and inflammation). The goal? To test combinations of these therapies to create the best “cocktail” for each patient.

Click on any drug in the interactive pipeline below to learn more about therapies in development for Duchenne.

Restoring or Replacing Dystrophin

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Dystrophin restoration or replacement aims to treat the underlying cause of Duchenne which is the lack of dystrophin, the protein that provides stability to the muscles. Exon skipping and nonsense mutation readthroughs are both ways that dystrophin restoration is being explored. Strategies to replace the missing dystrophin protein include gene therapy, which uses a modified smaller version of the dystrophin gene, called micro-dystrophin, to produce a modified micro-dystrophin protein.

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Combating Fibrosis

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Fibrosis, defined as the thickening and scarring of connective tissue, is a downstream symptom of the lack of dystrophin. Fibrosis occurs in Duchenne when chronic inflammation inhibits muscle repair. Reducing fibrosis may help decrease the breakdown of mature muscle cells and increase muscle strength.

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Reducing Inflammation

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Inflammation is a critical characteristic of Duchenne disease progression. Due to muscle degeneration and the resulting immune cells brought in to help regenerate the muscle, a whole host of inflammatory substances are released. The muscles of individuals with Duchenne are constantly in a state of inflammation. Corticosteroids are currently the standard of care to treat inflammation but have a number of side effects associated with long-term use. There are a number of experimental therapies in development that are aimed at reducing inflammation.

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Regulating Calcium Balance

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In Duchenne, because of the instability of the muscle membrane due to the lack of dystrophin, leaks in the muscle cell membrane can develop. These leaks can let too much calcium flow in and out of the muscle cell, disrupting cellular functions which further exacerbate cellular repair. Companies are developing compounds that aim to help regulate the calcium flow.

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Improving Muscle Growth & Protection

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Several therapeutic options intend to encourage muscle growth and discourage muscle breakdown. There are many strategies that can be explored in this domain, including approaches to enhance repair capabilities of the muscle, protect the muscle from breakdown, reduce inflammation and fibrosis, or induce muscle development.

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Restoring the Cells Energy

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Mitochondria are specialized structures within cells that supply chemical energy to power the activities of the cells, such as repairing muscle cells. Individuals with Duchenne lack efficient mitochondria in their cells. It is thought that by increasing or enhancing the mitochondria in muscle cells, the cellular functions of the muscle could be improved.

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Improving Heart Function

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Cardiac function is a concern in Duchenne, as the heart is a muscle and is affected by the lack of dystrophin. There are a number of therapies in development that may impact both the skeletal muscle and cardiac muscle. However, some strategies are aimed primarily at the heart. Use of known cardiac interventions – Angiotensin-converting-enzyme (ACE) inhibitors, Angiotensin receptor blockers (ARBs), Eplerenone, or Betablockers – are part of care management of the heart based on physician recommendation.

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