Gene therapy offers hope for kids with Hirschsprung disease [Video]

Danny is now 19 years old, but his mother, Amy, still remembers carrying him as an infant into the emergency room at Mass General for Children.”I was nursing him,” she said. “He started just spitting up and then throwing up. He didn’t want to eat, and he seemed distressed.”Dr. Allan Goldstein entered their room to examine Danny.”He pressed on his belly,” Amy said. “And his belly — even in the ER — started getting more and more distended, so I was freaking out. Like, ‘What is wrong?'”Goldstein knew the answer immediately.Danny had Hirschsprung disease.It occurs when nerve cells fail to develop in the large intestine.Without those cells, the large intestine is unable to detect or pass stool, leaving the bowel system paralyzed.”They get backed up,” Goldstein said. “They start to have vomiting or are unable to take food.”Danny’s parents were left with a life-altering decision to make for their son.He …