Cumberland Pharmaceuticals announced positive cardiac results from Phase 2 trial in Duchenne

Cumberland Pharmaceuticals announced positive cardiac results from Phase 2 trial in Duchenne
Cumberland Pharmaceuticals released positive top-line results from its Phase 2 FIGHT DMD trial, a 12-month placebo controlled study evaluating Ifetroban in 41 individuals with DMD. Individuals who received either low or high dose Ifetroban daily showed improvements in their heart’s left ventricular ejection fraction (LVEF), while the placebo cohort (as well as an external natural history control group) experienced a decline in LVEF during the same time span. Ifetroban is thought to work by blocking the thromboxane receptor, which has a role in inflammation and fibrosis.
What is Duchenne?
Duchenne Muscular Dystrophy, often abbreviated as DMD, is a progressive and severe muscle-wasting condition. Individuals diagnosed with DMD, a form of muscular dystrophy, face significant challenges. By the age of 12, many are reliant on wheelchairs due to muscle degeneration and also encounter social isolation. The condition complicates even the simplest daily activities and, as it progresses, critically affects the heart and respiratory muscles. Duchenne is classified under muscular dystrophies, genetic diseases marked by muscle weakness, with Duchenne being notably the most severe due to the absence of dystrophin, a protein essential for muscle integrity. Research into the genetic underpinnings of Duchenne has been ongoing since the dystrophin gene’s identification in 1986. CureDuchenne is committed to improving the lives of everyone affected through accelerating research to find a cure, improving care and empowering the our community.
Cardiac Dysfunction in Duchenne
Adolescence is typically when signs of cardiac dysfunction, a serious disease complication, first become apparent. Nonetheless, maintaining physical activity levels, even among those who are wheelchair-bound, can improve the management of cardiac symptoms.