Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group.

Köhn, Anja F; Grigull, Lorenz; du Moulin, Marcel; Kabisch, Sarah; Ammer, Luise; Rudolph, Cornelia; Muschol, Nicole M

Köhn, Anja F; Grigull, Lorenz; du Moulin, Marcel; Kabisch, Sarah; Ammer, Luise; Rudolph, Cornelia; Muschol, Nicole M.

Afiliação

Köhn AF; Department of Pediatrics, University Medical Center Hamburg Eppendorf, Hamburg, Germany.
Grigull L; Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany.
du Moulin M; Department of Pediatrics, University Medical Center Hamburg Eppendorf, Hamburg, Germany.
Kabisch S; Department of Pediatrics, University Medical Center Hamburg Eppendorf, Hamburg, Germany.
Ammer L; Department of Pediatrics, University Medical Center Hamburg Eppendorf, Hamburg, Germany.
Rudolph C; Department of Pediatrics, University Medical Center Hamburg Eppendorf, Hamburg, Germany.
Muschol NM; Department of Pediatrics, University Medical Center Hamburg Eppendorf, Hamburg, Germany.

Mol Genet Metab Rep ; 23: 100578, 2020 Jun.

Article em En | MEDLINE | ID: mdl-32226768

Palavras-chave

AEq, age-equivalent score; ATG, antithymocyte globulin; Avg., Average; DQ, developmental quotient; FPSS, four point scoring system; GAG, Glykosaminoglycans; HSCT; HSCT, hematopoietic stem cell transplantation; ICLD, International Center for Lysosomal Disorders; MPS IH, mucopolysaccharidosis type I (Hurler syndrome); MPS IIIA; MPS IIIA, mucopolysaccharidosis type IIIA; MPS IIIB, mucopolysaccharidosis type IIIB; Mucopolysaccharidosis type III; Natural history; SGSH, N-sulfoglucosamine sulfohydrolase; Sanfilippo syndrome; Stem cell transplantation; TDS, total disability score; UCBT, umbilical cord blood-derived hematopoietic stem cell transplantation; VABS-II, Vineland Adaptive Behavior Scales; y, years

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Ano de publicação: 2020 Tipo de documento: Article

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Ano de publicação: 2020 Tipo de documento: Article