Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease
Setting the Stage the Next Era of Gene Therapy for Ultrarare Disease https://pediatricsnationwide.org/wp-content/uploads/2025/12/Original_hi_res_JPG-091025SC0110_Color-for-web-1024x576.jpg 1024 576 Abbie Miller https://pediatricsnationwide.org/wp-content/uploads/2023/05/051023BT016-Abbie-Crop.jpg
Researchers at Nationwide Children’s Hospital recently administered a novel gene therapy targeting SLC6A1, advancing precision medicine for children who previously could not be treated. Maxwell, age 8, made history this fall, when he received a bespoke gene therapy targeting his ultrarare disease. This wasn’t the first time he worked with the team at Nationwide Children’s…