Spinraza
National Institutes of Health
Papers overview
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SPINRAZA demonstrated stabilization or improvement in motor function in individuals with spinal muscular atrophy (SMA) where the…
SMA is a rare hereditary neuromuscular disease that causes weakness and muscle wasting as a result of the loss of spinal motor…
Gene therapies featured prominently among this year's newsworthy drugs, some of which have already received a green light from…