Rimeporide
Rimeporide is an experimental drug for the treatment of Duchenne muscular dystrophy, being developed by the EspeRare foundation. it has been granted orphan drug status by the European Medicines Agency.Mechanism of action
The substance blocks an ion pump called sodium–hydrogen antiporter 1. While the exact mechanism is unknown, it is speculated that inhibition of this pump reduces sodium and calcium overload in cells of Duchenne patients.History
Rimeporide was designed as a treatment for chronic heart failure. It was unsuccessful in Phase I clinical trials, but was tolerated well by volunteers. Subsequently, the drug was sold to EspeRare, a Swiss nonprofit organisation that aims at developing drugs for rare diseases., the substance is in preclinical development for Duchenne.
