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Promising gene therapy delivers treatment directly to brain
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Rylae-Ann Poulin smiles as she learns to ride a horse in Bangkok, Thailand, Saturday, Jan. 14, 2023. Rylae-Ann was among the first to benefit from a new way of delivering gene therapy _ directly into the brain _ that experts believe holds great promise for treating a host of brain disorders. - photo by AP Photo/Sakchai Lalit
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.