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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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| Publicat a: | Ann Clin Transl Neurol |
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| Autors principals: | , , , , , , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
John Wiley and Sons Inc.
2020
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| Matèries: | |
| Accés en línia: | 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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