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Dyscorban for Duchenne muscular dystrophy

Last updated Aug. 28, 2025, by Margarida Maia, PhD
✅ Fact-checked by José Lopes, PhD

Administration
Clinical trials
Side effects

 

What is Dyscorban for Duchenne muscular dystrophy?

Dyscorban (ifetroban) is an oral treatment being developed by Cumberland Pharmaceuticals for cardiomyopathy — a general term for diseases of the heart muscle — associated with Duchenne muscular dystrophy (DMD).

DMD is a type of muscular dystrophy caused by mutations in the DMD gene, which encodes dystrophin, a protein that protects muscles during movement. Without it, muscles become damaged over time, leading to a gradual loss of muscle strength. Cardiomyopathy weakens the heart muscle, making it harder for the heart to pump blood.

The active ingredient in Dyscorban, ifetroban, is designed to block the thromboxane-prostanoid receptor. When this protein is activated, it makes heart muscle cells take in calcium, which can cause scarring, known as fibrosis, in the heart, and also lead to arrhythmias, or irregular heartbeats. By blocking it, Dyscorban is expected to reduce fibrosis and help preserve heart function.

Therapy snapshot

Treatment name: Dyscorban (ifetroban)
Administration: As a capsule by mouth
Clinical testing: In Phase 2 clinical testing for DMD

 

How will Dyscorban be administered?

Dyscorban is being tested in a Phase 2 clinical trial at a daily dose of 150 or 300 mg, given in the form of an oral capsule.

Dyscorban in clinical trials

Dyscorban is being tested in a fully enrolled Phase 2 clinical trial called FIGHT DMD (NCT03340675). A total of 48 men and boys with DMD, ages 7 and older, were randomly assigned to receive Dyscorban — at a daily dose of 150 or 300 mg — or a placebo for 12 months. After that one-year period, all patients entered an open-label extension to receive up to three years of treatment with high-dose Dyscorban.

Treatment was well tolerated and no serious side effects were reported, per early data. The left ventricle ejection fraction (LVEF) — a measure of how well the heart pumps blood — improved by an average of 1.8% in patients on high-dose Dyscorban, while it declined by 1.5% in those on the placebo, reflecting the typical worsening of heart function observed as DMD progresses. This combined for a difference of 3.3%.

When researchers added patients from a natural history study to the placebo group, the difference was even larger — by an average of 5.4%. High-dose Dyscorban also reduced biomarkers of heart damage, suggesting it may help protect the heart.

Dyscorban side effects

Side effects reported with Dyscorban during clinical testing included:

  • bruising
  • damage to small blood vessels

Muscular Dystrophy News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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This site is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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