Orphan Designation

An international group of scientists, led by Prof Gordon Lynch at the University of Melbourne, Australia, have shown that increasing the amount of HSP72 can have a beneficial effect in a mouse model of Duchenne muscular dystrophy. Giving the mice BGP-15 improved the function of the limb muscles and, more importantly, the breathing muscles as well as increasing the lifespan of the mice. The results were published in Nature magazine in 2011.

Data generated in the study suggest that BGP-15 may be effective in the treatment of Duchenne muscular dystrophy, therefore N-Gene has applied for and has been awarded orphan designation for BGP-15 in the treatment of DMD in the US and the European Union.