Childhood onset of Scheie syndrome, the attenuated form of mucopolysaccharidosis I.
(73/191)
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Effect of 6 years of enzyme replacement therapy on plasma and urine glycosaminoglycans in attenuated MPS I patients.
(74/191)
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Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.
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Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.
(76/191)
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Reference values for lysosomal enzymes activities using dried blood spots samples - a Brazilian experience.
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Histopathologic changes of the ear in canine models of mucopolysaccharidosis types I and VII.
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A tandem mass spectrometry triplex assay for the detection of Fabry, Pompe, and mucopolysaccharidosis-I (Hurler).
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Laronidase for cardiopulmonary disease in Hurler syndrome 12 years after bone marrow transplantation.
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