Antisense therapy delivers long-term correction of severe spinal muscular atrophy in mice

Scientists have discovered that the devastating neuromuscular disease, spinal muscular atrophy (SMA), might not exclusively affect the motor neurons in the spinal cord as has long been thought. Their study suggests that defects in peripheral tissues such as liver, heart, etc., might also contribute to SMA pathology in severely affected patients. The new findings also pave the way for a potential SMA drug to enter human trials by the end of the year.

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