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Drug treatment for spinal muscular atrophy type I
BACKGROUND: Spinal muscular atrophy (SMA) is caused by a homozygous deletion of the survival motor neuron 1 (SMN1) gene on chromosome 5, or a heterozygous deletion in combination with a point mutation in the second SMN1 allele. This results in degeneration of anterior horn cells, which leads to prog...
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| Publicado en: | Cochrane Database Syst Rev |
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| Autores principales: | , , , , , , |
| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
John Wiley & Sons, Ltd
2019
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| Acceso en línea: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6905354/ https://ncbi.nlm.nih.gov/pubmed/31825542 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/14651858.CD006281.pub5 |
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