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| Long-term clinical course of a patient with mucopolysaccharidosis type IIIB |
| Ja Hye Kim, Yang Hyun Chi, Gu-Hwan Kim, Han-Wook Yoo, Jun Hwa Lee |
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Clin Exp Pediatr. 2016;59(Suppl 1):S37-S40. Published online November 30, 2016 DOI: https://doi.org/10.3345/kjp.2016.59.11.S37 |
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Long-term clinical course of a patient with mucopolysaccharidosis type IIIB Korean Journal of Pediatrics. 2016;59(Suppl 1):S37  Long-Term Follow-up Posthematopoietic Stem Cell Transplantation in a Japanese Patient with Type-VII Mucopolysaccharidosis Diagnostics. 2020;10(2):105 Molecular genetics of mucopolysaccharidosis type IIIA and IIIB: Diagnostic, clinical, and biological implications Human Mutation. 2001;18(4):264-281 Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis type IV A Molecular Genetics and Metabolism. 2014;111(2):S104 Hemiatrophy of the Spinal Cord in a Patient With Mucopolysaccharidosis Type IIIB Journal of Child Neurology. 2010;25(10):1288-1291 Intracerebral gene therapy for mucopolysaccharidosis type IIIB syndrome The Lancet Neurology. 2017;16(9):681-682 871. Long-Term Expression of Sleeping Beauty Transposon in the Murine Model of Mucopolysaccharidosis (MPS) Type VII Molecular Therapy. 2004;9:S331 Delivery of adenine base editors to patient-derived induced pluripotent stem cells in vitro: A putative treatment for mucopolysaccharidosis type IIIB Molecular Genetics and Metabolism. 2019;126(2):S39 Induced pluripotent stem cell line (SDQLCHi041-A) from a male patient with mucopolysaccharidosis type IIIB Stem Cell Research. 2021;52:102212 identification of pharmacological chaperones for mucopolysaccharidosis type IIIB Molecular Genetics and Metabolism. 2020;129(2):S101 |
