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34247932 | A multicenter open-label extension study of intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A. | 2021 Sep-Oct |
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30528227 | Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: A phase IIb randomized trial. | 2019 Feb |
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31385193 | Bioinformatics classification of mutations in patients with Mucopolysaccharidosis IIIA. | 2019 Dec |
1 |
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29251941 | Reduction in Brain Heparan Sulfate with Systemic Administration of an IgG Trojan Horse-Sulfamidase Fusion Protein in the Mucopolysaccharidosis Type IIIA Mouse. | 2018 Feb 5 |
1 |
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23274385 | Mild mental retardation and low levels of urinary heparan sulfate in a patient with the attenuated phenotype of mucopolysaccharidosis type IIIA. | 2013 May |
2 |
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23863627 | Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. | 2013 Aug 1 |
1 |
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21963444 | Helper-dependent canine adenovirus vector-mediated transgene expression in a neurodegenerative lysosomal storage disorder. | 2012 Jan 1 |
3 |
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22547151 | Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA mice. | 2012 Aug |
2 |
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21910976 | Molecular characterization of MPS IIIA, MPS IIIB and MPS IIIC in Tunisian patients. | 2011 Nov 20 |
1 |
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20231109 | SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors. | 2010 Jun |
2 |
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20370351 | Genetic manipulation of murine embryonic stem cells with enhanced green fluorescence protein and sulfatase-modifying factor I genes. | 2010 May |
1 |
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20673764 | Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice. | 2010 Oct |
2 |
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18407553 | The mutation p.Ser298Pro in the sulphamidase gene (SGSH) is associated with a slowly progressive clinical phenotype in mucopolysaccharidosis type IIIA (Sanfilippo A syndrome). | 2008 May |
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18410855 | Dilated perivascular spaces: an informative radiologic finding in Sanfilippo syndrome type A. | 2008 May |
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16828069 | Characterization of a C57BL/6 congenic mouse strain of mucopolysaccharidosis type IIIA. | 2006 Aug 9 |
2 |
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16846011 | Directed differentiation and characterization of genetically modified embryonic stem cells for therapy. | 2006 |
1 |
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15146460 | Transport, enzymatic activity, and stability of mutant sulfamidase (SGSH) identified in patients with mucopolysaccharidosis type III A. | 2004 Jun |
3 |
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11793481 | Sanfilippo syndrome in Turkey: Identification of novel mutations in subtypes A and B. | 2002 Feb |
1 |
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11509012 | Prediction of Sanfilippo phenotype severity from immunoquantification of heparan-N-sulfamidase in cultured fibroblasts from mucopolysaccharidosis type IIIA patients. | 2001 Aug |
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