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Do we always need to treat patients with spinal muscular atrophy? A personal view and experience

BACKGROUND: We report the clinical outcomes observed in our patients with SMA type 1 or 2 receiving nusinersen, and we comment on the ethical implications of this treatment, in line with our results and those reported by Audic et al. in their analysis published in the Orphanet Journal of Rare Diseas...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Agosto, Caterina, Salamon, Eleonora, Divisic, Antuan, Benedetti, Francesca, Giacomelli, Luca, Shah, Aashni, Perilongo, Giorgio, Benini, Franca
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2021
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7879688/
https://ncbi.nlm.nih.gov/pubmed/33573692
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01593-4
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