Advances in Pediatric Gene Therapy for Neuromuscular Disorders

5/29/2024; 39 minutes

As part of the 2024 Developmental Disabilities Conference, Dr. Peter Kang, Professor of Genetics at the University of Minnesota, presents a detailed summary of recent advances in gene therapy for children with neuromuscular disorders, specifically Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, and Pompe Disease. He uses case studies to demonstrate diagnostic dilemmas, intricacies of developing and delivering treatment, and the importance of shared decision making about therapies with families. Recorded on 03/08/2024. (#39747)

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