Promising gene therapy delivers treatment directly to brain

Rylae-Ann Poulin attends physical education class at Ruamrudee International School in Bangkok, Thailand, Friday, Jan. 13, 2023. The extremely rare AADC deficiency disorder afflicts about 135 children worldwide, many in Taiwan. Judy Wei, who was born in Taiwan, and her husband Richard Poulin III, sought out a doctor there who correctly diagnosed Rylae-Ann. They learned she could qualify for a gene therapy clinical trial in Taiwan. (AP Photo/Sakchai Lalit)