Early Development of Spinal Deformities in Children Severely Affected with Spinal Muscular Atrophy after Gene Therapy with Onasemnogene Abeparvovec—Preliminary Results

Spinal muscular atrophy (SMA) is a rare genetic disorder, with the most common form being 5q SMA. Survival of children with severe SMA is poor, yet major advances have been made in recent years in pharmaceutical treatment, such as gene-therapy, which has improved patient survival. Therefore, clinica...

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Detalles Bibliográficos
Autores principales: Soini, Venla, Schreiber, Gudrun, Wilken, Bernd, Hell, Anna Kathrin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10297104/
https://www.ncbi.nlm.nih.gov/pubmed/37371230
http://dx.doi.org/10.3390/children10060998

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10297104/
https://www.ncbi.nlm.nih.gov/pubmed/37371230
http://dx.doi.org/10.3390/children10060998

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