Infusion of gene therapy into the brain ameliorates an uncommon childhood disorder.

A group of thirty children suffering from AADC deficiency have shown improvement in their terrible symptoms after receiving gene-replacement treatment.

Some infants who were born with an extremely uncommon genetic ailment have been given the ability to walk and talk for the very first time thanks to a gene-replacement therapy that is given straight to the brain.

Richard Poulin, whose daughter Rylae-Ann underwent the therapy when she was 18 months old in November 2019, said, “It's a dream come true.” Rylae-Ann was treated. A woman named Rylae-Ann who lives in Thailand went from being unable to utter a single word, move, or even lift her head to being able to “run, leap, kick a ball, ride a horse, swim, and communicate in numerous languages,”…

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