Protective Effects of Butyrate-based Compounds on a Mouse Model for Spinal Muscular Atrophy

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Butchbach, Matthew E. R. ; Lumpkin, Casey J. ; Harris, Ashlee W. ; Saieva, Luciano ; Edwards, Jonathan D. ; Workman, Eileen ; Simard, Louise R. ; Pellizzoni, Livio ; Burghes, Arthur H. M. (2016)

Proximal spinal muscular atrophy (SMA) is a childhood-onset degenerative disease resulting from the selective loss of motor neurons in the spinal cord. SMA is caused by the loss of SMN1 (survival motor neuron 1) but retention of SMN2. The number of copies of SMN2 modifi... View more
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