Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma)

Spinal Muscular Atrophy (SMA) is a genetic disease that causes weakness and wasting in the voluntary muscles of infants and children. SMA has been the leading inherited cause of infant death. More specifically, SMA is caused by the absence of the SMN1 gene. In May 2019, the Food and Drug Administrat...

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Detalles Bibliográficos
Autores principales: Ogbonmide, Tolu, Rathore, Rajni, Rangrej, Shahid B, Hutchinson, Stedrea, Lewis, Marcia, Ojilere, Stephenie, Carvalho, Victoria, Kelly, Irenaissia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Cureus 2023
Materias:
Genetics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10104684/
https://www.ncbi.nlm.nih.gov/pubmed/37065340
http://dx.doi.org/10.7759/cureus.36197

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10104684/
https://www.ncbi.nlm.nih.gov/pubmed/37065340
http://dx.doi.org/10.7759/cureus.36197

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