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...
Guardado en:
Autores principales: | Andreia Lomba, Joana A. Ribeiro, Henriqueta Araújo, Vera Ribeiro, Núria Madureira, Filipe Palavra, Carmen Costa, Isabel Fineza |
---|---|
Formato: | article |
Lenguaje: | EN PT |
Publicado: |
Sociedade Portuguesa de Pediatria
2021
|
Materias: | |
Acceso en línea: | https://doaj.org/article/828fb533814c45c68d07e54b9f60eae3 |
Etiquetas: |
Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!
|
Ejemplares similares
-
Improvement in Fine Manual Dexterity in Children with Spinal Muscular Atrophy Type 2 after Nusinersen Injection: A Case Series
por: Minsu Gu, et al.
Publicado: (2021) -
Intrathecal Administration of Nusinersen Using the Ommaya Reservoir in an Adult with 5q-Related Spinal Muscular Atrophy Type 1 and Severe Spinal Deformity
por: Vasileios Papaliagkas, et al.
Publicado: (2021) -
Modeling spinal muscular atrophy in Drosophila.
por: Howard Chia-Hao Chang, et al.
Publicado: (2008) -
Paediatric dermatological conditions in an emergency department: a single-centre study in Thailand
por: Rattapon Uppala, et al.
Publicado: (2021) -
Healthcare professionals’ perspectives on infant feeding support in paediatric inpatients: single-centre qualitative study
por: Elizabeth Evans, et al.
Publicado: (2021)