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Successful weaning from mechanical ventilation in a patient with SMA type 1 treated with nusinersen

SMA type 1 is the most severe type, characterized by early onset at <6 months of age, and rapid progression resulting in permanent assisted ventilation before 2 years of life. Supportive care was the only treatment until the approval of nusinersen, an antisense oligonucleotide drug that increases...

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Detalles Bibliográficos
Autores principales: Lee, Jiwon, Park, Se Eun, Lee, Dajeong, Song, Joo Young, Lee, Jeehun
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8045896/
https://www.ncbi.nlm.nih.gov/pubmed/33616311
http://dx.doi.org/10.1002/acn3.51321