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...
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| Format: | Article |
| Language: | English |
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MDPI AG
2018-08-01
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| Series: | Journal of Clinical Medicine |
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| Online Access: | http://www.mdpi.com/2077-0383/7/9/251 |