• Promising gene therapy delivers treatment directly to brain

    11 days ago - By STAT

    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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