Used in research for the treatment of Sanfilippo syndrome (Mucopolysaccharidosis type A), a rare genetic disorder characterized by the accumulation of heparan sulfate due to enzyme deficiency. Acts as a pharmacological chaperone to stabilize the defective enzyme, enhancing its activity and promoting lysosomal function. Investigated for its ability to cross the blood-brain barrier, making it a candidate for addressing neurological symptoms associated with the disease. Primarily utilized in preclinical studies to evaluate substrate reduction therapy and long-term cognitive outcomes.