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CSF GAG reduction is associated with a decrease in inflammationMOLECULAR GENETICS AND METABOLISMIF3.52025-02-010PRE
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Identification of inflammatory cells in dilated ascending aortas of IDUA-deficient (MPS I) miceMOLECULAR GENETICS AND METABOLISMIF3.52024-02-010PRE
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Is time growth? The impact of early initiation and duration of enzyme replacement therapy on growth in Hurler syndromeMOLECULAR GENETICS AND METABOLISMIF3.52024-02-010PRE
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Hurler Syndrome Glycosaminoglycans Decrease in Cerebrospinal Fluid without Brain-Targeted TherapyANNALS OF NEUROLOGYIF7.72023-09-231OAAI
Hematopoietic cell transplantation for mucopolysaccharidosis type I in the presence of decreased cardiac functionMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Long-term intravenous galsulfase in Maroteaux-Lamy syndrome further reduces urine glycosaminoglycans (GAG) after hematopoietic stem cell transplantation and improves cardiac function and enduranceMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
Decreases in CSF neuro-inflammatory markers are associated with gain in neurocognitive function after ERT plus HCT in Hurler syndromeMOLECULAR GENETICS AND METABOLISMIF3.52023-02-010PRE
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Contribution of the innate and adaptive immune systems to aortic dilation in murine mucopolysaccharidosis type IMOLECULAR GENETICS AND METABOLISMIF3.52022-03-014OAAI
Aortic dilation in murine mucopolysaccharidosis type I: A tale of two strainsMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Bone marrow and umbilical cord blood are equivalent stem cell sources for Hurler syndromeMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
Treatment of cardiac, neurologic, and skeletal manifestations of murine MPS I with AAV9-IDUA: Efficacy study of vector dose and route of administrationMOLECULAR GENETICS AND METABOLISMIF3.52022-02-010PRE
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