Spinal Muscular Atrophy

Nusinersen Improves Motor Function, Survival in Infants With Spinal Muscular Atrophy

Nusinersen Improves Motor Function, Survival in Infants With Spinal Muscular Atrophy

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Patients who received nusinersen had a greater likelihood of survival compared to those who received sham treatment.

FDA Approves First Treatment for Spinal Muscular Atrophy

FDA Approves First Treatment for Spinal Muscular Atrophy

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The US FDA has approved the first drug for the treatment of spinal muscular atrophy, a genetic disease that often results in premature death.

New Drug for Spinal Muscular Atrophy Shows Promise

New Drug for Spinal Muscular Atrophy Shows Promise

Despite the benefits observed, the improvements still do not represent normal muscle function.

Gene Therapy Stops, Stabilizes Spinal Muscular Atrophy in Animal Model

Gene Therapy Stops, Stabilizes Spinal Muscular Atrophy in Animal Model

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The study was the first to model the disease in a large animal with similar physiology to humans.

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