Families battle muscle-wasting disease
Two Ridgefield families are in the thick of the fight against Duchenne muscular dystrophy, a progressive muscle-wasting genetic condition that steals movement and shortens young lives. Now one of the families has been thrust into the thick of a fight for a seat on the U.S. Supreme Court.
Jessica and Chris Curran’s 10-year-old son Conner, who has been battling Duchenne muscular dystrophy for five years, was highlighted Monday by U.S. Sen. Richard Blumenthal D-Conn., who told Conner’s story to demonstrate the importance of the Affordable Care Act — “Obamacare,” which the Trump administration has challenged in court — in relation to the nomination of Judge Amy Coney Barrett to the highest court in the land.
“Conner is a superhero, but he’s always had a real sidekick,” Blumenthal told the Senate Judiciary Committee hearing on Judge Barrett’s nomination. “He’s had the protection of the Affordable Care Act. It has shielded him and his family from arbitrary caps on coverage that would have cut off his care when it became too expensive. It has protected Conner from losing insurance because of this insidious disease that he never caused or chose.
“The Affordable Care Act has given his family a measure of relief, of hope, of peace. They still worry about Conner’s health, but not their coverage or its costs,” Blumenthal said.
“Conner, and millions of others like him, are why I will oppose your nomination.”
A champion
Duchenne muscular dystrophy (DMD) is a pre-existing condition and without the Affordable Care Act, his family and Blumenthal fear the 10-year-old “could lose access to critical care.”
“For us, if Conner was ever denied health care coverage because of his pre-existing condition, our family would be in a terrible situation,” Chris Curran said. “We work hard, but we’d
never be able to afford care for Conner without insurance.”
Blumenthal has met with the family about once a year to discuss ACA and see how Conner is doing. The disease is considered a “fatal illness” that requires “tens of of thousands of dollars in medical treatment every year” Blumenthal’s office said.
Duchenne muscular dystrophy affects mostly young boys. A genetic condition, it is marked by the progressive loss of muscle as the body fails to make dystrophin, a muscle building protein. It affects many parts of the body, with deterioration of the skeletal, cardiac, and pulmonary muscles. Some 90 percent of boys with DMD become non-ambulatory in their early teenage years, and many don’t live past their late 20s.
“With Duchenne’s, it’s a progressive muscular disease,” said Jessica Curran. “It’s a steady, predictable decline. These boys, they lose their muscles in their legs and then they lose their muscles in their arms … Then the heart and lungs...
“They have special care they need,” she said.
Team Teddy
Among the “millions of others” Blumenthal referred to as fighting the disease is Ridgefielder Kate and Justin White’s son, Teddy. Nearing the age of 3, he has been
diagnosed with Duchenne muscular dystrophy.
In support, 14 women ran 5K and 10K courses through Ridgefield’s hills on Saturday, Oct. 3, raising about $9,000 for the fight against the Duchenne’s muscular dystrophy.
“They were all Ridgefield moms, all local friends of mine,” Kate White said. “We wanted to keep it really small with COVID precautions.”
They didn’t do any advance publicity.
“It was really just through word of mouth,” she said.
A committed runner who ran the New York Marathon in 2010, Kate White had been training to run in the Chicago Marathon this year as a fundraiser to help battle the disease. Covid-19 put an end to that plan as the marathon was called off. But she decided to run locally, and soon had a group of women joining her to support her son’s fight.
They became Team Teddy. “Everyone was so into it, and ‘You have to do this again, we’d love to make it a year thing, if we could,’” she said.
“This January I decided to run the 2020 Chicago Marathon in order to raise awareness and support for research to find a cure for Duchenne muscular dystrophy (DMD) , a devastating disorder that my youngest son,
Teddy had recently been diagnosed with,” Kate White wrote. “Friends Bonnie Pope, Emily Carr and Rachel Marino had planned to join me prior to the cancellation due to COVID.
“Although the marathon may have been canceled, the race to end DMD is still going strong! ‘Team Teddy’ has raised over $8,000 from friends, family, and complete strangers, and we were given the chance to run Chicago once over the next three years.”
Kate White’s “Race to End Duchenne’ fundraising website, which can be found online.
Ardent supporters
Of all the supporters White and her son have had, among the most ardent and helpful have been the Currans.
“We would not get through this without the help of Jessica and Chris Curran,” White said.
“They live essentially a mile from us. Their kids go to Barlow, as well. They’ve been so supportive of us,” she said.
The Curran’s fund-raising website “Kindness Over Muscular Dystrophy” (https://www.kindnessovermd.org/) offers a heartfelt portrait of their son.
“Conner has the kind of smile that lights up any room and brings joy to everyone he meets,” it says. “Beneath this beaming smile is an immensely brave little boy who is quite literally in a fight for his life.
“This terrible disorder makes muscles so weak that it takes away the ability of a child to walk, to hug, to hold hands, and eventually even robs them of their smile,” it reads. “Conner’s giant smile has been the constant from the time he was born. Whether playing with his brothers in the snow or sand, exploring the world, or even more recently, through clinical trials, MRIs and constant poking and prodding … always that smile. “
“When his friends or brothers run around outside and he needs to watch and wait, still that smile.
“As Conner’s mother Jess explains it, ‘I will always carry him, always fight for him and take care of him, but I cannot smile for him.’
“The Duchenne diagnosis shattered the Currans and brought them to their knees,” the website says. “As parents, they would have traded places with Conner in a heartbeat to save him from the pain that would certainly come if no cure is found.
“Any thoughts of a typical childhood for Conner vanished. There would be no soccer, no basketball, no baseball, no lacrosse... but really, the sports mean nothing. Walking at the age of 12 means something.”
Gene therapy
The new gene therapy raising hopes for Conner Curran and kids like him was highlighted in July by National Public Radio.
The NPR piece quoted Jessica Curran recalling advice she’d gotten from the doctor who diagnosed Conner in 2015: “Take your son home, love him, take him on trips while he’s walking, give him a good life and enjoy him because there are really not many options right now.”
Conner was the first child to receive the experimental gene treatment — a single infusion designed to fix the genetic mutation that was gradually causing his muscle cells to die, NPR said. While the treatment couldn’t bring back the cells he’d lost, it allowed his remaining muscle cells to function better.
In an interview Monday, Chris Curran explained some of the treatment’s limits, and the family’s hopes.
“We are hopeful that the treatment he received will last,” Conner’s father said. “The scientists predict this treatment could last anywhere from five to seven years — for Duchenne’s that’s a miracle.”
The genes used in the therapy are delivered into the body using a virus. And while that was successful for the first treatment, the body reacts to the virus and creates antibodies so the viral delivery system can’t be used a second time — at least not at the current stage of research and scientific understanding.
“But it bought us time, which in Duchenne’s is a miracle. It may have bought us five years for science to catch up with how we re-dose successfully,” he said.