Type-1 spinal muscular atrophy cohort before and after disease-modifying therapies
Both groups showed improvement in motor function and stabilization of respiratory and bulbar function, with the difference between the groups possibly being related to the earlier treatment initiation. Thus, the present study provides valuable insights into the real-world safety and clinical efficacy of disease-modifying therapies for SMA 1 patients.
Authors
- Flávia Nardes (ORCID: https://orcid.org/0000-0001-8006-9435)
- Márcia Gonçalves Ribeiro (ORCID: https://orcid.org/0000-0001-8906-0189)
- Alexandra Prufer de Queiroz Campos Araújo (ORCID: https://orcid.org/0000-0003-2591-4354)
- Brenda Alves (ORCID: https://orcid.org/0009-0002-9429-1875)
Institutions
- Universidade Federal do Rio de Janeiro (BR)
- Universidade Federal do Estado do Rio de Janeiro (BR)
Publication Details
- Journal
- Arquivos de Neuro-Psiquiatria
- Published
- 2024-11-01
- DOI
- https://doi.org/10.1055/s-0044-1791757
- Citations
- 4
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.75