Lysosomal storage diseases (LSDs) are about 50 rare inherited metabolic disorders that result from defects in lysosomal function. They are characterized by an abnormal build-up of various toxic materials in the body’s cells due to enzyme deficiencies. New lysosomal storage disorders continue to be identified. While clinical trials are in progress on possible treatments for some of these diseases, there is currently no approved treatment for many lysosomal storage diseases.

Our mission is to improve people’s lives with rare diseases, and we are dedicating our efforts to 3 different Lysosomal Storage Disorders: Alpha mannosidosis, Nephropathic cystinosis, and Fabry disease.