Teaching an old drug new tricks: repositioning strategies for spinal muscular atrophy
(2019)
Journal Article
Bowerman. (2019). Teaching an old drug new tricks: repositioning strategies for spinal muscular atrophy. Future Neurology, 14(3), https://doi.org/10.2217/fnl-2019-0006
Spinal muscular atrophy (SMA) is a childhood disorder caused by loss of the survival motor neuron (SMN) gene. Pathological hallmarks are spinal cord motor neuron death, neuromuscular junction dysfunction and muscle atrophy. The first SMN genetic ther... Read More about Teaching an old drug new tricks: repositioning strategies for spinal muscular atrophy.