Nusinersen in type 0 spinal muscular atrophy: should we treat?

A male infant affected by type 0 SMA with one copy of SMN2 received early treatment with Nusinersen at the age of 13 days. He showed mild motor improvement 2 months after treatment started but despite also showing some minimal respiratory improvement, required tracheostomy at the age of 4 months and...

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Detalles Bibliográficos
Publicado en:Ann Clin Transl Neurol
Autores principales: Tiberi, Eloisa, Costa, Simonetta, Pane, Marika, Priolo, Francesca, de Sanctis, Roberto, Romeo, Domenico, Tiziano, Francesco D., Conti, Giorgio, Vento, Giovanni, Mercuri, Eugenio
Formato: Artigo
Lenguaje:Inglês
Publicado: John Wiley and Sons Inc. 2020
Materias:
Case Study
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7732235/
https://ncbi.nlm.nih.gov/pubmed/33147378
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.51126
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