Treatment for Spinal Muscular Atrophy

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Abstract

In a randomized, double-blind, sham-controlled study of infants with spinal muscular atrophy (SMA) type 1, nusinersen treatment led to significant improvement in motor milestones and survival without significant adverse reactions. A safety trial of gene therapy for SMA type 1 demonstrated prolonged ventilator-free survival and improved motor milestones with minimal side effects.

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