Nusinersen: Single-Centre Real-Life Experience in Type 1 Spinal Muscular Atrophy
Introduction: Nusinersen, an antisense oligonucleotide designed to treat spinal muscular atrophy, led to substantial motor milestone achievements in clinical trials. The aim of this study was to report the clinical outcome of children diagnosed with spinal muscular atrophy type 1 treated with nusin...
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| Autores principales: | , , , , , , , |
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| Formato: | article |
| Lenguaje: | EN PT |
| Publicado: |
Sociedade Portuguesa de Pediatria
2021
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| Materias: | |
| Acceso en línea: | https://doaj.org/article/828fb533814c45c68d07e54b9f60eae3 |
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