Gene therapy infused into the brain eases rare condition in children

A gene-replacement therapy has eased the debilitating symptoms in a group of 30 children with AADC deficiency


27 September 2022

Rylea-Ann was unable to walk before receiving the gene therapy

Judy Wei

A gene-replacement therapy that is administered directly to the brain has allowed some children with a rare genetic condition to walk and talk for the first time.

“It’s a dream come true,” says Richard Poulin, whose daughter Rylae-Ann received the therapy in November 2019, aged 18 months. Rylae-Ann, who lives in Thailand, went from being unable to say any words, move or even lift her head to “running, jumping, kicking a ball, riding a horse, swimming and speaking in multiple languages”, …

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