Cargando…

Muscle: an independent contributor to the neuromuscular spinal muscular atrophy disease phenotype

Spinal muscular atrophy (SMA) is a pediatric-onset neuromuscular disorder caused by insufficient survival motor neuron (SMN) protein. SMN restorative therapies are now approved for the treatment of SMA; however, they are not curative, likely due to a combination of imperfect treatment timing, inadeq...

Descripción completa

Detalles Bibliográficos
Autores principales: Jha, Narendra N., Kim, Jeong-Ki, Her, Yoon-Ra, Monani, Umrao R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10561723/
https://www.ncbi.nlm.nih.gov/pubmed/37737261
http://dx.doi.org/10.1172/jci.insight.171878