Divergent clinical outcomes of alpha-glucosidase enzyme replacement therapy in two siblings with infantile-onset Pompe disease treated in the symptomatic or pre-symptomatic state.

Matsuoka, Takashi; Miwa, Yoshiyuki; Tajika, Makiko; Sawada, Madoka; Fujimaki, Koichiro; Soga, Takashi; Tomita, Hideshi; Uemura, Shigeru; Nishino, Ichizo; Fukuda, Tokiko; Sugie, Hideo; Kosuga, Motomichi; Okuyama, Torayuki; Umeda, Yoh

Matsuoka, Takashi; Miwa, Yoshiyuki; Tajika, Makiko; Sawada, Madoka; Fujimaki, Koichiro; Soga, Takashi; Tomita, Hideshi; Uemura, Shigeru; Nishino, Ichizo; Fukuda, Tokiko; Sugie, Hideo; Kosuga, Motomichi; Okuyama, Torayuki; Umeda, Yoh.

Afiliação

Matsuoka T; Showa University Northern Yokohama Hospital, Children Medical Center, Yokohama, Kanagawa, Japan.
Miwa Y; Showa University Northern Yokohama Hospital, Children Medical Center, Yokohama, Kanagawa, Japan.
Tajika M; Showa University Northern Yokohama Hospital, Children Medical Center, Yokohama, Kanagawa, Japan.
Sawada M; Showa University Northern Yokohama Hospital, Children Medical Center, Yokohama, Kanagawa, Japan.
Fujimaki K; Showa University Northern Yokohama Hospital, Children Medical Center, Yokohama, Kanagawa, Japan.
Soga T; Showa University Northern Yokohama Hospital, Children Medical Center, Yokohama, Kanagawa, Japan.
Tomita H; Showa University Northern Yokohama Hospital, Cardiovascular Center, Yokohama, Kanagawa, Japan.
Uemura S; Showa University Northern Yokohama Hospital, Cardiovascular Center, Yokohama, Kanagawa, Japan.
Nishino I; Department of Neuromuscular Research, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo, Japan.
Fukuda T; Department of Pediatrics, Hamamatsu University School of Medicine, Hamamatsu, Shizuoka, Japan.
Sugie H; Faculty of Health and Medical Sciences, Tokoha University, Hamamatsu, Shizuoka, Japan.
Kosuga M; Division of Medical Genetics, National Center for Child Health and Development, Setagaya-ku, Tokyo, Japan.
Okuyama T; Department of Clinical Laboratory Medicine, National Center for Child Health and Development, Setagaya-ku, Tokyo, Japan.
Umeda Y; Showa University Northern Yokohama Hospital, Children Medical Center, Yokohama, Kanagawa, Japan.

Mol Genet Metab Rep ; 9: 98-105, 2016 Dec.

Article em En | MEDLINE | ID: mdl-27896132

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Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Diagnostic_studies Idioma: En Revista: Mol Genet Metab Rep Ano de publicação: 2016 Tipo de documento: Article País de afiliação: Japão

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