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mucopolysaccharidosis type iiia
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Assay of heparan-N-sulfamidase in dried leukocytes impregnated in filter paper: a new tool for the i…
BMP4, SGSH, and SLC11A2 are Predicted to Be Biomarkers of Aging Associated with Programmed Cell Deat…
Is SGSH heterozygosity a risk factor for early-onset neurodegenerative disease?
Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysacchar…
An Engineered sgsh Mutant Zebrafish Recapitulates Molecular and Behavioural Pathobiology of Sanfilip…
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Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS …
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidos…
Natural History Studies of Mucopolysaccharidosis III
Natural History Study of Patients With MPS IIIA
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