Highlights Summary Reduced survival motor neuron (SMN) protein triggers the motor neuron disease, spinal muscular atrophy (SMA). Restoring SMN prevents disease, but it is not
Earlier this year, the US Food and Drug Administration approved the most expensive drug ever to hit the market, a gene therapy for spinal muscular
Biomedical researchers tackling genetic diseases dream of tracing a clean, straight line from discovering a mutation to understanding a disease’s mechanism to developing a curative
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