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Therapeutics development for spinal muscular atrophy
Spinal muscular atrophy is an autosomal recessive motor neuron disease that is the leading inherited cause of infant and early childhood mortality. Spinal muscular atrophy is caused by mutation of the telomeric copy of the survival motor neuron gene (SMN1), but all patients retain a centromeric copy...
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| Formato: | Artigo |
| Idioma: | Inglês |
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Springer-Verlag
2012
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| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3593434/ https://ncbi.nlm.nih.gov/pubmed/16554261 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nurx.2006.01.010 |
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