A rare disease affects only a small percentage of the population, the reason why they have been neglected for years.

Yet, a common mechanism is shared by many: an inherited mutation that ultimately results in the destabilization of a protein.

Our goal is to tackle this common pathogenic mechanism with the design of disease-specific pharmacological chaperones.

This strategy may be able to correct the phenotype for a plethora of rare syndromes. To that end, we use Chassys™, an NMR based innovative and efficient proprietary Platform Technology approach.