The association between mutations in the lysosomal protein glucocerebrosidase and parkinsonism. (1/208)

Continuous infusion of enzyme replacement therapy is inferior to weekly infusions in MPS I dogs. (2/208)

Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression. (3/208)

Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease. (4/208)

Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis. (5/208)

Type 2 Gaucher disease occurs in Ashkenazi Jews but is surprisingly rare. (6/208)

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. (7/208)

Converting an injectable protein therapeutic into an oral form: phenylalanine ammonia lyase for phenylketonuria. (8/208)

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