Preliminary insights into RNA in CSF of pediatric SMA patients after 6 months of nusinersen

BACKGROUND: Spinal muscular atrophy (SMA) is a rare autosomal-recessive neurodegenerative disorder caused by mutations in survival motor neuron 1 (SMN1) gene, and consequent loss of function of SMN protein, which results in progressive loss of lower motor neurons, and muscular wasting. Antisense oli...

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Detalles Bibliográficos
Autores principales: Garofalo, M., Bonanno, S., Marcuzzo, S., Pandini, C., Scarian, E., Dragoni, F., Di Gerlando, R., Bordoni, M., Parravicini, S., Gellera, C., Masson, R., Dosi, C., Zanin, R., Pansarasa, O., Cereda, C., Berardinelli, A., Gagliardi, S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10498611/
https://www.ncbi.nlm.nih.gov/pubmed/37705059
http://dx.doi.org/10.1186/s13062-023-00413-6

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10498611/
https://www.ncbi.nlm.nih.gov/pubmed/37705059
http://dx.doi.org/10.1186/s13062-023-00413-6

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