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Patients Thought Untreatable with Rare Disease Dramatically Improve with Common Gene Therapy

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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,…

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News Source: www.goodnewsnetwork.org

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