GDNF-based therapy for Hirschsprung disease

Hirschsprung disease (HSCR) is a deadly birth defect affecting 1/5000 children. These children are missing innervation from the enteric nervous system (ENS), resulting in severe constipation and a high risk of bacterial translocation into blood causing premature death. Surgery to remove ENS-devoid bowel is generally life-saving but many children with HSCR continue to have severe problems after surgery.