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Orphanet J Rare Dis. 2021 Jul 15;16(1):312. doi: 10.1186/s13023-021-01935-w.
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
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Bow Hunter's Syndrome.
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Stroke. 2022 Jan;53(1):e26-e29. doi: 10.1161/STROKEAHA.121.037253. Epub 2021 Dec 6.
Molecular characterization of 355 mucopolysaccharidosis patients reveals 104 novel mutations.
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Gene therapy for Mucopolysaccharidoses.
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Glycan-based biomarkers for mucopolysaccharidoses.
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Mol Genet Metab. 2014 Feb;111(2):73-83. doi: 10.1016/j.ymgme.2013.07.016. Epub 2013 Jul 29.
Mucopolysaccharidosis IVA and glycosaminoglycans.
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The diagnosis and management of mucopolysaccharidosis type II.
Mao SJ, Chen QQ, Dai YL, Dong GP, Zou CC.
Ital J Pediatr. 2024 Oct 8;50(1):207. doi: 10.1186/s13052-024-01769-9.
Neurobehavioral phenotypes of neuronopathic mucopolysaccharidoses.
Barone R, Pellico A, Pittalà A, Gasperini S.
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