Gene Therapy PTC-AADC Provides Benefits for 5 Years in Children

Gene Therapy PTC-AADC Provides Benefits for 5 Years in Children

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Gene Therapy PTC-AADC Provides Benefits for 5 Years in Children

The investigational gene therapy PTC-AADC (eladocagene exuparvovec) provides lasting and meaningful improvements in motor and cognitive function in children with aromatic l-amino acid decarboxylase (AADC) deficiency, updated results from three clinical trials show. The greatest improvements were observed in the youngest of the 26 treated children — none of whom had full head control at the study’s start — with three of them gaining the ability to walk freely without assistance following gene therapy. Another little…

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