Rimeporide: safety, tolerability and pharmacokinetic results from a phase Ib study in DMD boys as well as exploratory biomarkers

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In Duchenne muscular dystrophy, impaired sodium and calcium handling are associated with severe osmotic oedema, muscle weakness, inflammation and fibrosis. Rimeporide, a safe and potent NHE-1 inhibitor, represents a novel target for DMD patients. Rimeporide is an orally available drug showing to promote compelling cardioprotective effect in the dystrophic hamsters with dilated cardiomyopathy. In mdx mice Rimeporide demonstrated promising data on skeletal muscle function, fibrosis and inflammation in skeletal, cardiac and diaphragm muscles.

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