Each day more are being discovered. The majority of rare diseases do not have even a single FDA approved treatment. Glycomine is developing therapeutics for diseases that have no treatment options. We are driven to develop therapies that provide significant improvements in the quality of life for patients and families with rare diseases.
We focus on genetic disorders of protein and lipid glycosylation, which often cause severe debilitation in patients. There are 100 known glycosylation disorders. Glycomine is currently addressing two disorders of glycosylation: PMM2-CDG and Ngly1 deficiency.
Glycomine's therapeutic approach is to utilize replacement therapies — substrates, enzymes, or proteins — depending upon the disease target and clinically relevant organ system.