Newborn screening for spinal muscular atrophy in Japan: One year of experience

Spinal muscular atrophy (SMA) is a degenerative neuromuscular disease that causes progressive muscle weakness and atrophy due to loss of the anterior horn cells of the spinal cord. Although effective treatments, such as gene therapy, have emerged in recent years, their therapeutic efficacy depends o...

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Detalles Bibliográficos
Autores principales: Sawada, Takaaki, Kido, Jun, Sugawara, Keishin, Yoshida, Shinichiro, Ozasa, Shiro, Nomura, Keiko, Okada, Kentaro, Fujiyama, Natsumi, Nakamura, Kimitoshi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9356196/
https://www.ncbi.nlm.nih.gov/pubmed/35942129
http://dx.doi.org/10.1016/j.ymgmr.2022.100908

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