Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome).

Poswar, Fabiano de Oliveira; Henriques Nehm, Johanna; Kubaski, Francyne; Poletto, Edina; Giugliani, Roberto

Poswar, Fabiano de Oliveira; Henriques Nehm, Johanna; Kubaski, Francyne; Poletto, Edina; Giugliani, Roberto.

Afiliação

Poswar FO; Clinical Research Group in Medical Genetics, Clinical Research Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.
Henriques Nehm J; Medical Genetics Service, Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil.
Kubaski F; Postgraduate Program in Genetics and Molecular Biology, UFRGS, Porto Alegre, RS, Brazil.
Poletto E; DR Brasil Research Group, HCPA, Porto Alegre, RS, Brazil.
Giugliani R; Clinical Research Group in Medical Genetics, Clinical Research Center, Hospital de Clínicas de Porto Alegre, Porto Alegre, RS, Brazil.

Ther Clin Risk Manag ; 18: 1143-1155, 2022.

Article em En | MEDLINE | ID: mdl-36578769

Palavras-chave

Sly syndrome; enzyme replacement therapy; gene therapy; hematopoietic stem cell transplantation; lysosomal disorders; mucopolysaccharidosis type VII

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Tipo de estudo: Diagnostic_studies Idioma: En Revista: Ther Clin Risk Manag Ano de publicação: 2022 Tipo de documento: Article

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