Promising gene therapy delivers treatment directly to brain

About 30 U.S. studies are testing gene therapy delivered directly to the brain for various disorders, including AADC deficiency as well as Alzheimer’s, Parkinson’s and Huntington’s.

When Rylae-Ann Poulin was a year old, she didn’t crawl or babble like other kids her age. A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep.

Then, months later, doctors delivered gene therapy directly to her brain.

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