Advances and limitations for the treatment of spinal muscular atrophy

Spinal muscular atrophy (5q-SMA; SMA), a genetic neuromuscular condition affecting spinal motor neurons, is caused by defects in both copies of the SMN1 gene that produces survival motor neuron (SMN) protein. The highly homologous SMN2 gene primarily expresses a rapidly degraded isoform of SMN prote...

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Detalles Bibliográficos
Autores principales: Day, John W., Howell, Kelly, Place, Amy, Long, Kimberly, Rossello, Jose, Kertesz, Nathalie, Nomikos, George
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9632131/
https://www.ncbi.nlm.nih.gov/pubmed/36329412
http://dx.doi.org/10.1186/s12887-022-03671-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9632131/
https://www.ncbi.nlm.nih.gov/pubmed/36329412
http://dx.doi.org/10.1186/s12887-022-03671-x

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