Patients Thought Untreatable with Rare Disease Dramatically Improve with Common Gene Therapy
A lumbar puncture – credit, BruceBlaus CC 3.0. via Wikimedia Commons A single-dose gene replacement therapy is found to transform the capabilities for movement in children over 2 years of age and teenagers with spinal muscular atrophy, according to research published in Nature Medicine. The effects allowed these minors who could sit but not stand … Read more