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 the disease which is the lack of dystrophin, the protein that provides stability to the muscles. Gene therapy, exon skipping, and nonsense mutation readthroughs are all ways that dystrophin restoration/replacement is being explored.

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Combating Fibrosis

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Fibrosis, defined as the thickening and scarring of connective tissue, usually as a result of injury, is a downstream symptom of the lack of dystrophin and occurs as chronic inflammation prevents 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 cells brought in to help regenerate the muscle, namely immune cells, 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 treatment to treat inflammation but have a number of side effects associated with use. There are a number of experimental therapies companies are developing 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 can develop. These leaks 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. One of the most common targets for drug development with this strategy is restraining a protein called myostatin. In healthy muscle, myostatin helps to maintain muscle at a reasonable size. In Duchenne, with loss of muscle, myostatin further destabilizes the muscle. Researchers hope that blocking myostatin may allow the muscles to grow and be more stable. Companies are developing therapies aimed at inhibiting (restraining) myostatin.

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Restoring the Cells Energy

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Mitochondria are powerhouse organelles — the specialized structures within living cells that supply chemical energy to drive the activities of the cells, including repairing muscle cells. Individuals with Duchenne have dysfunctional mitochondria, which in turn inhibits muscle repair. It is thought that by increasing or enhancing the mitochondria in muscle cells, that muscle repair 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. It is thought that many therapeutic strategies will impact the heart, if they impact the skeletal 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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