rimeporide

Rimeporide is an orally available small molecule drug that acts as a first-in-class inhibitor of the sodium–hydrogen exchanger type 1 (NHE-1). Originally developed for advanced congestive heart failure, it has been repositioned for Duchenne muscular dystrophy (DMD) due to its cardioprotective and anti-fibrotic properties. Rimeporide works by selectively inhibiting NHE-1, a membrane protein responsible for regulating intracellular pH and sodium levels. Inhibition of NHE-1 helps reduce pathological calcium overload in muscle cells—a key contributor to muscle damage in DMD—by correcting sodium and pH imbalances. The drug has demonstrated safety and tolerability in Phase 1b clinical trials involving boys with DMD, showing promising effects on cardiac and skeletal muscle function. Rimeporide is being developed by EspeRare Foundation, which licensed it from Merck Serono[1][3][4][5][6].