Combination Therapy with Nusinersen and Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy Type I

Background: Spinal muscular atrophy (SMA) is a neuromuscular progressive disease, characterized by decreased amounts of survival motor neuron (SMN) protein, due to an autosomal recessive genetic defect. Despite recent research, there is still no cure. Nusinersen, an antisense oligonucleotide acting...

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Detalles Bibliográficos
Autores principales: Mirea, Andrada, Shelby, Elena-Silvia, Axente, Mihaela, Badina, Mihaela, Padure, Liliana, Leanca, Madalina, Dima, Vlad, Sporea, Corina
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8658131/
https://www.ncbi.nlm.nih.gov/pubmed/34884240
http://dx.doi.org/10.3390/jcm10235540

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8658131/
https://www.ncbi.nlm.nih.gov/pubmed/34884240
http://dx.doi.org/10.3390/jcm10235540

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