Promising early gene therapy trial for ‘childhood dementia’

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An investigational gene therapy for a syndrome that leads to a form of childhood dementia has shown promising early results in a proof-of-concept study, a conference will hear today.

The study into Sanfilippo syndrome by researchers at Manchester University NHS Foundation Trust found after being given OTL-21, an investigational gene therapy, four out of five patients gained cognitive skills in line with development in healthy children.