Development and Translation of Therapies for Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterised by widespread loss of lower motor neurons from the spinal cord, leading to progressive weakness and muscle atrophy. SMA is largely caused by homozygous loss of the survival motor neuron (SMN) 1 gene, resulti...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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European Medical Journal
2016-07-01
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| Series: | European Medical Journal Neurology |
| Subjects: | |
| Online Access: | http://emjreviews.com/wp-content/uploads/Development-and-Translation-of-Therapies-for-Spinal-Muscular-Atrophy.pdf |