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New Directions for SMA Therapy
Spinal muscular atrophy (SMA) is a severe disorder of motor neurons and the most frequent genetic cause of mortality in childhood, due to respiratory complications. The disease occurs due to mutations in the survival motor neuron 1 (SMN1) gene that leads to a reduction in the SMN protein, causing de...
Gespeichert in:
| Veröffentlicht in: | J Clin Med |
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| 1. Verfasser: | |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
MDPI
2018
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6162810/ https://ncbi.nlm.nih.gov/pubmed/30200278 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jcm7090251 |
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