Gene Therapy Success Story: LAD-1 Siblings Thrive | JQJO

Three siblings, Ava, Olivia, and Landon Langenhop, suffered from the rare LAD-1 disease, causing constant infections and hospitalizations. After exhausting other options, they participated in a UCLA clinical trial using gene therapy. The experimental treatment, developed by Rocket Pharmaceuticals, successfully produced healthy immune cells, eliminating severe infections. The therapy is now under FDA review, offering hope for future LAD-1 patients. The children are thriving five years post-treatment, attending school and participating in activities.