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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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Molecular characterization of 355 mucopolysaccharidosis patients reveals 104 novel mutations.
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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.
The diagnosis and management of mucopolysaccharidosis type II.
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Genotype-phenotype findings in patients with mucopolysaccharidosis II from the Hunter Outcome Survey.
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Mucopolysaccharidosis IVA and glycosaminoglycans.
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Therapies for the bone in mucopolysaccharidoses.
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