Newborn screening and gene therapy in SMA: Challenges related to vaccinations

Spinal muscular atrophy (SMA) affects one in 7,500–10,000 newborns. Before the era of disease-modifying therapies, it used to be the major genetic cause of mortality in infants. Currently, there are three therapies approved for SMA, including two molecules modifying the splicing of the SMN2 gene and...

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Detalles Bibliográficos
Autores principales: Kotulska, Katarzyna, Jozwiak, Sergiusz, Jedrzejowska, Maria, Gos, Monika, Ogrodnik, Magdalena, Wysocki, Jacek, Czajka, Hanna, Kuchar, Ernest
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Neurology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9727226/
https://www.ncbi.nlm.nih.gov/pubmed/36504644
http://dx.doi.org/10.3389/fneur.2022.890860

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9727226/
https://www.ncbi.nlm.nih.gov/pubmed/36504644
http://dx.doi.org/10.3389/fneur.2022.890860

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