BOULDER, Colo. — A Boulder girl’s family has been able to raise enough money to schedule a clinical trial for their daughter’s rare disease but now they need to raise $1 million by September to pay for the trial drug.
In December, Mila Makovec was diagnosed with Batten disease, a neurodegenerative disease. It’s fatal and many children die before they reach their teens.
Five years ago, doctors said there wasn’t much hope for people with Batten disease, but now doctors think a cure is possible. With hope on the horizon, the family is trying to launch a clinical trial to find answers.
“We want to play our role. We want to help our daughter. We want to help our son if that’s what it comes down to and we want to be able to help other families,” said Julia Vitarello, Mila’s mom.
Her family has raised $1 million to get the science going and have another $1 million committed. The gene therapy trial is schedule for the spring at a leading hospital.
However, the family said without an additional $1 million by September to pay for the trial drug, everything stops. The family has launched a GoFundMe page to raise the additional money.