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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...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2021
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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 |