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Nusinersen: A Review in 5q Spinal Muscular Atrophy
Survival motor neuron 1 (SMN1), located on chromosome 5q, encodes the survival motor neuron (SMN) protein. A deletion or mutation in SMN1 results in a rare neuromuscular disorder: 5q spinal muscular atrophy (SMA). In such patients, SMN protein production relies solely on SMN2. Nusinersen (Spinraza(®...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Springer International Publishing
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8709816/ https://www.ncbi.nlm.nih.gov/pubmed/34850360 http://dx.doi.org/10.1007/s40263-021-00878-x |