At Minoryx, we are committed to finding innovative treatments for life threatening rare diseases. We focus on pediatric diseases and we are currently working on treatments for neurometabolic diseases of genetic origin.

Minoryx develops a new generation of small molecule drugs known as pharmacological chaperones, which offer the most promising approach to the treatment of genetic diseases severy affecting the central nervous system. As a complementary approach, Minoryx is also involved in repositioning-based projects.