BRANFORD, Conn. (WTNH) — When you first met the Herzogs in December 2019, they were starting a high-tech journey to help their son, Max, who suffers from duchenne muscular dystrophy.
“I keep referring to it as a scientific miracle because it is a miracle,” said mom, Stephanie.
This past January, the first step completed. A muscle specimen was taken from the 5-year-old. Using CRISPR technology, experts edit the gene mutation in a lab, hoping to correct the problem.
“As soon as they edited that gene mutation, his cells just immediately auto-corrected and started producing dystrophin as they should,” explained Stephanie, noting that’s the desired outcome to halt the progression of the fatal neuromuscular condition that impacts boys.
But another big procedure is on the horizon.
“Transporting those corrected cells back into the body to get the body to produce a majority of corrected cells,” said dad, Jesse.
“Time is the greatest enemy here,” said Rich Horgan, who founded the organization Cure Rare Disease and is behind the scientific push, in honor of his brother, Terry. He’s continually working with the FDA to advance this customized therapy. “We’re all fighting against time and fighting against this faceless disease that continues to rob Terry and Max and too many other boys.”
“We’ll start to move into mouse models, animal models, for safety, so above all, we do no harm,” he said
The pandemic has presented fundraising challenges but Horgan has tapped into a creative solution – using gaming events to raise money.
“These guys, boys, can’t go outside and play basketball but they can whoop my butt in a game of Fortnite,” he said.
It’s all to keep hope for a healthy future alive.
“To have the opportunity for us to dream and to let them dream about what they can be, I can’t even tell you how valuable it is,” said Stephanie.
If all goes well, the plan is for Max’s next procedure to take place in early 2022.
More information about the potentially groundbreaking therapy can be found online.