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13-1445C What A Cure Looks Like?

Speaker Discussion Panel

Existing and developing technologies offer a broad range of approaches to intervention in genetic disease. Options include: dietary restriction or supplementation, small molecule inhibitors or effectors, enzyme replacement, stem cell therapy, organ or tissue transplant, gene editing or therapy, and others. Different approaches may be better suited to different conditions depending on the timing and severity of outcomes, the nature of the genetic defect, and key organs impacted, among other factors. Here the speakers will share their thoughts about the pros and cons of different options for intervention in classic galactosemia.