Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of spinal cord motor neurons, muscle atrophy and infantile death or severe disability. It is caused by severe reduction of the ubiquitously expressed survival motor neuron (SMN) protein, due to loss of the SM...
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| Format: | Journal article |
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Oxford University Press
2017
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