Gene therapy for spinal muscular atrophy is considerably effective when administered as early as possible after birth.

Sawada, Takaaki; Kido, Jun; Yae, Yukako; Yuge, Kotaro; Nomura, Keiko; Okada, Kentaro; Fujiyama, Natsumi; Ozasa, Shiro; Nakamura, Kimitoshi

Sawada, Takaaki; Kido, Jun; Yae, Yukako; Yuge, Kotaro; Nomura, Keiko; Okada, Kentaro; Fujiyama, Natsumi; Ozasa, Shiro; Nakamura, Kimitoshi.

Afiliación

Sawada T; Southern Kyushu and Okinawa Unit Center of The Japan Environment and Children's Study, Faculty of Life Sciences, Kumamoto University, Kumamoto, Japan.
Kido J; Department of Pediatrics, Faculty of Life Sciences, Kumamoto University, Kumamoto, Japan.
Yae Y; Department of Pediatrics, Kumamoto University Hospital, Kumamoto, Japan.
Yuge K; Department of Pediatrics, Faculty of Life Sciences, Kumamoto University, Kumamoto, Japan.
Nomura K; Department of Pediatrics, Kumamoto University Hospital, Kumamoto, Japan.
Okada K; Department of Pediatrics and Child Health, Kurume University School of Medicine, Fukuoka, Japan.
Fujiyama N; Department of Pediatrics and Child Health, Kurume University School of Medicine, Fukuoka, Japan.
Ozasa S; Department of Pediatrics, Kumamoto University Hospital, Kumamoto, Japan.
Nakamura K; Department of Pediatrics, Kumamoto University Hospital, Kumamoto, Japan.

Mol Genet Metab Rep ; 35: 100973, 2023 Jun.

Article en En | MEDLINE | ID: mdl-37091744

Palabras clave

Gene therapy; Newborn screening; Onasemnogene abeparvovec; SMN1; Spinal muscular atrophy

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Genet Metab Rep Año: 2023 Tipo del documento: Article País de afiliación: Japón Pais de publicación: Estados Unidos

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