MPS I Archives - MPS Society

Overcoming limitations inherent in sulfamidase to improve MPS IIIA gene therapy

Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disease caused by the deficiency of sulfamidase. MPS IIIA is characterized by progressive neurodegeneration accompanied by loss of social skills and … Continued

Validation of small molecule therapeutic leads for treatment of MPS I disease

Mucopolysaccharidosis I (MPS I) is a serious genetic disease that leads to early death. In this disease, there is a single enzyme that is missing or deficient, an enzyme that … Continued