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New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges
Spinal muscular atrophy (SMA) is one of the most common autosomal recessive diseases with progressive weakness of skeletal and respiratory muscles, leading to significant disability. The disorder is caused by mutations in the survival motor neuron 1 (SMN1) gene and a consequent decrease in the SMN p...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2020
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7408870/ https://www.ncbi.nlm.nih.gov/pubmed/32668756 http://dx.doi.org/10.3390/jcm9072222 |