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Is there hope for spinal muscular atrophy synthetic pharmacotherapy? |
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| Authors: | Markus Riessland |
| Institution: | Laboratory of Molecular and Cellular Neuroscience, The Rockefeller University, New York, NY, USA |
| Abstract: | |
| Keywords: | Antisense oligonucleotides (ASO) nusinersen spinal muscular atrophy spinal cord survival of motor neuron (SMN2) splicing correction therapy |