In the Pipeline-Spinal Muscular Atrophy: Gene Therapy Found Effective for Children with SMA – LWW Journals
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In the Pipeline-Spinal Muscular Atrophy: Gene Therapy Found Effective for Children with SMA
LWW Journals Fifteen boys with spinal muscular atrophy 1 have reached developmental motor milestones, previously not achievable, with gene therapy to replace defective spinal motor neuron 1. Independent experts said the results are encouraging, but questions remain … |
