Evaluation of the Efficacy of Nusinersen Treatment in Patients with Late-onset SMA Using the Hammersmith Functional Motor Scale Expanded
Objective: Spinal muscular atrophy (SMA) is a hereditary disorder with progressive muscle weakness and atrophy. Nusinersen is an antisense oligonucleotide directed against SMN2 and has been shown in studies to improve the motor skills of patients. The aim of this study was to evaluate the efficacy o...
| Principais autores: | , , , , , |
|---|---|
| Formato: | Artigo |
| Idioma: | English |
| Publicado em: |
Galenos Publishing House
2024-08-01
|
| coleção: | Journal of Behçet Uz Children's Hospital |
| Assuntos: | |
| Acesso em linha: | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=behcetuz&plng=eng&un=BUCHD-28000 |