MN child with rare disease continues to receive support, gene therapy possible
A family from the southern metropolis is about to have their two-year-old child participate in the first gene therapy of its kind, while the community works with them to raise funds.
We first told you about Lucy Hieb and her journey with CTNNB1 syndrome in early 2023 – back then, her family raised more than $100,000 to support the CTNNB1 Foundation in researching and investigating a possible cure.
CTNNB1 is a rare nervous system disorder with only about 500 confirmed cases worldwide. Lucy is one of them. According to her family, the disorder affects her ability to walk, talk and even think.
Now they’re back at the fundraising table as the foundation is about to begin a clinical trial. On Wednesday, Lucy’s Lemonade Stand in Lakeville raised $7,500 – their goal this time is $20,000.
“We want to do everything we can to give her the best possible chance at a better future,” said Megan Hieb, Lucy’s mother.
This included a trip to Slovenia this summer, where the CTNNB1 Foundation is headquartered and held its second ever conference.
“I think we left feeling really empowered and encouraged and full of hope,” said Charlie Hieb, Lucy’s father.
At the conference they were informed about the progress and next steps of the possible clinical trial.
“A team of doctors screened 83 children with Lucy syndrome for possible participation in the gene therapy clinical trial,” Megan said of her journey.
That clinical trial could begin as early as next summer and will only enroll 10 to 15 children—there’s no guarantee Lucy will make it into the first round. Still, they’re working to raise money, as the Hieb family says the foundation’s budget could run out in December.
“This disease is so rare that it’s not profitable. So it’s up to the families, we kind of have the responsibility to raise funds and make this possible,” Megan said.
You can support Lucy on her journey by donating online – an important note from the family: click on the “Donate” button and select “Gene Therapy Research” to ensure you are supporting this specific project.
