Author Archives: Yedida Y Bogachkov PhD

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…