An infant with type II Gaucher disease treated with enzyme replacement therapy.

Ebihara, Go; Matsushita, Yuki; Kirino, Makiko; Hikino, Shunji; Sato, Kazuo

Ebihara, Go; Matsushita, Yuki; Kirino, Makiko; Hikino, Shunji; Sato, Kazuo.

Afiliación

Ebihara G; Department of Pediatrics, Clinical Research Institute, Kyushu Medical Center, National Hospital Organization, Fukuoka, Japan.
Matsushita Y; Department of Pediatrics, Clinical Research Institute, Kyushu Medical Center, National Hospital Organization, Fukuoka, Japan.
Kirino M; Department of Pediatrics, Clinical Research Institute, Kyushu Medical Center, National Hospital Organization, Fukuoka, Japan.
Hikino S; Department of Pediatrics, Clinical Research Institute, Kyushu Medical Center, National Hospital Organization, Fukuoka, Japan.
Sato K; Department of Pediatrics, Clinical Research Institute, Kyushu Medical Center, National Hospital Organization, Fukuoka, Japan.

Pediatr Int ; 64(1): e14873, 2022 Jan.

Article en En | MEDLINE | ID: mdl-34847281

Asunto(s)

Enfermedad de Gaucher; Terapia de Reemplazo Enzimático; Enfermedad de Gaucher/tratamiento farmacológico; Glucosilceramidasa; Humanos; Lactante; Resultado del Tratamiento

Palabras clave

L483R; enzyme replacement therapy; quality of life; type II Gaucher disease

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedad de Gaucher Aspecto: Patient_preference Límite: Humans / Infant Idioma: En Revista: Pediatr Int Asunto de la revista: PEDIATRIA Año: 2022 Tipo del documento: Article País de afiliación: Japón

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