Hope, Beyond Measure: Ridgefield Family Stares Down Rare Disease

RIDGEFIELD, CT — Ten-year-old Conner Curran hit all his pediatric milestones, right up until about six years ago. That's when his parents noticed he was severely falling behind his twin brother, Kyle.

Then began the battery of doctor visits, hospital stays and tests that all parents dread, followed by a prognosis which was the one parents dread even more.

The Barlow Elementary 4th grader suffers from Duchenne muscular dystrophy, a particularly rare, insidious and progressive disease that over time robs children of all their muscle functions.

The genetic disorder affects about one in 5,000 boys, and sneaks in somewhere around age four — round about the first time he's picking up a baseball bat or a musical instrument. Legs are the first to go, and most patients will end up in a wheelchair around the time they are 10-years-old.

"They lose their arm muscles in their teen years, where they can't feed themselves or take care of themselves, or even hug," Jessica explained. "And they lose their lives, mid-20s."

"Our world went to pieces. We were in the darkest place of our lives, getting that diagnosis."

Yet, as Jessica recalled it, even that diagnosis didn't hold a candle to the grim pep talk delivered by the genetics specialist as their son was being released:

"Y'know, just take Conner home, give him a good life, take him on vacations while he's walking, because nobody survives this, and there really isn't any hope."

That cruel proclamation of a slow-motion death sentence for their son should have been devastating, and crippling, for the Currans, but they instead took it as a challenge.

"We have a very big faith in God, and science, and it didn't make sense that this was happening in our lives," Jessica said.

The couple created a foundation, Kindness Over Muscular Dystrophy, to raise money for research, as well as advocate in Washington D.C., and help other families purchase vital medical and accessibility equipment such as power wheelchairs, chair lifts and accessible vans.

As they fell deeper down the pharmaceutical business rabbit hole, they began to understand that the powerful forces of the market were arrayed against them. Throw a few million dollars towards finding a treatment for, say, the coronavirus, and you'll still reap a healthy profit. But a successful treatment for a disease like Duchenne, borne of a genetic mutation, may not even work for the next case. What pharmaceutical company is going to spend millions on research for a cure that may only help one person?

Rich Horgan had asked himself that question years earlier while a student at Harvard Business School. His brother, Terry, was also born with Duchenne, and was approaching his mid-20s.

The answer to the question, Horgan deduced, was to form a group of researchers and clinicians with the mission of developing custom therapeutics for patients who had genetic diseases that were too rare to attract pharmaceutical interest.

That organization, Cure Rare Disease, founded three years ago and based in Boston, is funded by "literally hundreds of thousands of donors" that are contributing one-, five- and ten-dollar donations to the cause. It would be easier to wrangle fewer and larger donors, Horgan told Patch, but that would miss the point of his grassroots effort.

"The idea is to have a non-profit biotech company that is fueled in a way that really is able to prioritize the patient, rather than the profits."

The organization will be holding its annual "Community for a Cure" fundraiser on June 5 in Stamford, and hopes to raise $250,000.

The Currans met Rich Horgan last summer at a charity golf tournament and "were blown away by what he is doing."

Cure Rare Disease is using CRISPR gene-editing technology to re-tool genetic errors, customizing therapeutics for children with Duchenne and other rare diseases.

Shortly after meeting Horgan, Jessica joined the board of Cure Rare Disease.

"I needed a seat at the table," she said. "That's how much I believe in Cure Rare Disease. They are going to change the face of Duchenne."

The organization is in the final stages of preparation before administering its first custom therapeutic for Duchenne — on Terry — this fall.

Conner has a bit more time, but the clock is still ticking, his race is a bit longer. But now, according to his mother, it's a race he has a chance of winning:

"This has given me hope beyond measure."