At the end of this session, learners will be able to gain an overview of genetic diagnostic advances and its therapeutic implications in neuromuscular diseases, review disease modifying therapies (gene therapy and RNA based therapies) for spinal muscular atrophy (SMA), and discuss gene-targeted therapy for Duchenne muscular dystrophy (DMD).
This seminar was delivered at the 16th SEHA International Pediatric Conference on March 25-27, 2022.
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