Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II.

Seo, Joo-Hyun; Kosuga, Motomichi; Hamazaki, Takashi; Shintaku, Haruo; Okuyama, Torayuki

Seo, Joo-Hyun; Kosuga, Motomichi; Hamazaki, Takashi; Shintaku, Haruo; Okuyama, Torayuki.

Afiliação

Seo JH; Department of Clinical Laboratory Medicine, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan.
Kosuga M; Department of Clinical Laboratory Medicine, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan.
Hamazaki T; Department of Pediatrics, Osaka City University Graduate School of Medicine, 1-4-3 Asahi-machi, Abeno-ku, Osaka 545-8585, Japan.
Shintaku H; Department of Pediatrics, Osaka City University Graduate School of Medicine, 1-4-3 Asahi-machi, Abeno-ku, Osaka 545-8585, Japan.
Okuyama T; Department of Clinical Laboratory Medicine, National Center for Child Health and Development, 2-10-1 Okura, Setagaya-ku, Tokyo 157-8535, Japan.

Mol Ther Methods Clin Dev ; 21: 67-75, 2021 Jun 11.

Article em En | MEDLINE | ID: mdl-33768130

Palavras-chave

Kyoto Scale of Psychological Development 2001 (KSPD); developmental age; enzyme replacement therapy; heparan sulfate; idursulfase beta; infusions; intracerebroventricular; mucopolysaccharidosis II; pediatrics; psychology

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Texto completo: 1 Bases de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Japão