Dorphan, a company dedicated to the development of drugs for rare and orphan diseases

Dorphan S.A. is a drug Discovery and development company committed to the development of innovative therapeutics for the treatment of rare and devastating lysosomal storage diseases with high unmet medical need

Lysosomal storage diseases are a group of inherited metabolic disorders caused by the deficiency of lysosomal functions, in most cases due to genetic defects in the lysosomal enzymes responsible for the elimination of cellular constituents. In patients affected by lysosomal storage diseases the accumulation over time of a variety of substrates in multiple tissues and organs causes irreversible damages.

Our mission is to develop innovative therapies, including pharmacological chaperones, with the objective to rescue lysosomal enzyme function and improve patients’ life.  

 


June 2022

Dorphan Announces FDA Grant of Orphan Drug Designation for DO-10 in the Treatment of GM1-gangliosidosis.