Family fights to cure 3-year-old son
Gala to aid boy with rare, debilitating muscle condition
The Handt family’s lives changed forever late last year.
Jennifer and Rick Handt’s 3-year-old son, Charlie, was diagnosed with a rare, debilitating and potentially fatal muscle condition: Duchenne muscular dystrophy.
It was then that the couple began their fight for Charlie’s future.
“We have learned to embrace every day … and that is so much easier with such a wonderful community supporting us and Charlie,” Jennifer Handt said. “Every single day we are doing something to help Charlie. We have a good life, and there is so much hope on the horizon.”
The Handt family, and the families of other children with Charlie’s diagnosis, are gathering June 5 for a gala benefit to help fund a cure. The goal: $250,000.
Duchenne is a genetic condition that typically impacts males and leads to muscle deterioration. A progressive condition, most children who get it will need to use a wheelchair by their early teens, with heart and breathing issues sometimes to follow.
“Currently our son has some symptoms,” Jennifer Handt said, “but is still mobile.”
The Handt family has spent months learning about the condition and the potentially life-changing gene therapy that may lie ahead.
“The science is evolving so quickly … there is so much cause for hope,” Jennifer Handt said.
The key for further research remains funding, which led the Handts and other Connecticut families with children suffering from DMD to team with Cure Rare Disease, a nonprofit biotechnology research organization on a mission to develop precision medicine for rare diseases.
“Community for a Cure,” which will celebrate progress made in developing customized therapeutics and raise funds to support patient research underway at Yale School of Medicine, will hold its early June benefit at The Village in Stamford.
“This will be a special evening for the Cure Rare Disease family to come together to celebrate our recent progress and to demonstrate our commitment to the mission of curing rare disease,” Rich Horgan, founder and president of Cure Rare Disease, said.
Horgan said Cure Rare Disease is approaching the final stages of developing a gene therapy treatment for DMD. Horgan’s brother, Terry, was born with DMD and will be the first person to receive the treatment later this year, he said.
“I am extremely grateful to all of our sponsors, donors and families who support and champion our work and keep us steadily on the path of progress toward our goal of saving lives,” Horgan said.
The $250,000 goal the organization has set will be earned — with luck — during a hybrid in-person and virtual program featuring both a silent and a live auction with professional auctioneer Harry Santa-Olalla. Among the prizes: an exclusive dinner for eight at Darien Butcher Shop’s “underground restaurant,” The Charles.
Signed copies of Walter Isaacson’s book “The Code Breaker” will be given at the Champion level. The book highlights the Nobel Prize-winning inventor of CRISPR, the specific type of gene therapy that is the basis of the new treatment for DMD being used in the Yale trial.
The event will include an awards ceremony recognizing clinical and scientific contributions.
For the Handts, the celebration of clinical progress in the fight against Duchenne muscular dystrophy is another sign of hope that further funding can accelerate work on the final cure to save Charlie, they said.
“Charlie is the happiest child,” Jennifer Handt said. “His smile just lights up a room … he loves life, loves his family. But I know underneath his muscles are withering away every day. Every parent in this situation would do what we are doing — fighting for a cure.”
For more information about the gala’s sponsorship and donation opportunities and to purchase tickets, visit https://givergy.us/ CRDCommunityForACureGala/ ?controller=home.
For more information on the Handts’ Charlie’s Cure fund with Fairfield County’s Community Foundation, visit curecharlie.org.