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

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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Bibliographic Details
Main Authors: Katarzyna Kotulska, Sergiusz Jozwiak, Maria Jedrzejowska, Monika Gos, Magdalena Ogrodnik, Jacek Wysocki, Hanna Czajka, Ernest Kuchar
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-11-01
Series:Frontiers in Neurology
Subjects:
spinal muscular atrophy
newborn screening
vaccinations
gene therapy
onasemnogene abeparvovec
Online Access:https://www.frontiersin.org/articles/10.3389/fneur.2022.890860/full

Internet

https://www.frontiersin.org/articles/10.3389/fneur.2022.890860/full

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