Spinal Muscular Atrophy
Patients who received nusinersen had a greater likelihood of survival compared to those who received sham treatment.
The US FDA has approved the first drug for the treatment of spinal muscular atrophy, a genetic disease that often results in premature death.
Despite the benefits observed, the improvements still do not represent normal muscle function.
The study was the first to model the disease in a large animal with similar physiology to humans.
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