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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society for Clinical Investigation
2023
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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 |