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Gene Therapies for Neuromuscular Conditions
Objectives:
- Understand gene therapy in the neuromuscular arena
- Principles
- Mechanism of action
- Efficacy
- Side effects
- Management
- Spinal muscular atrophy and Duchenne muscular dystrophy
- Genetics and pathophysiology
- Natural history
- Forward thinking
- Consider what diseases could be a candidate for gene transfer and why
- Begin to speculate as to “second generation” of gene transfer
Randal Richardson, MD, MMS, Pediatric Neuromuscular Neurologist and Director of the Muscular Dystrophy Association Clinic at Gillette Children’s, presents information about the use of gene therapies for the neuromuscular
