Scientists say delivering gene therapy directly to the brain holds great promise. The first brain-delivered gene therapy on the market was recently approved in Europe and the U.K. for a rare genetic disorder called AADC deficiency.
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.
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. Then, months later. doctors delivered gene therapy directly to her brain.
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