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From Bench to Bedside: Gene Therapy Approaches for Rare Diseases

Gene replacement therapy is emerging as a potential treatment for rare CNS diseases. In this recorded CME satellite symposium, a parent of a child with spinal muscular atrophy describes their journey through gene therapy clinical trials. Using spinal muscular atrophy as an example, three faculty experts walk through the history of gene therapy from the bench to the bedside.
9829-0001