Family using crowdfunding to help find cure for daughter's rare disease

- What would you do if doctors told you your child had an incurable disease that would take their life before they became a teenager?

One local family is facing this heartbreaking scenario and they say they are willing to do anything to help their 7-year-old girl -- even if that means raising millions of dollars on their own to try and find a cure.

Ellie McGinn, of Arlington, dreams of becoming a singer in Hollywood or an artist in Paris. And her parents are determined to help her get there even though the second grader was diagnosed at the age of three with leukodystrophy or LBSL.

The degenerative brain and spinal cord disease is so rare that Ellie is one of less than 100 cases ever identified. The genetic condition has no cure.

"We have to think about the future not worrying about it,” her mother Beth McGinn said. “We just have to appreciate that she is here today and she’s such an awesome kid that it makes it easy.”

An experimental cocktail of prescription vitamins and enzymes has stopped Ellie's pain and is preventing seizures. But since she is the first person in the world with LBSL to ever take it, nobody knows how long it will work. Most with this disease are in wheelchairs by the age of seven and die before they are teenagers.

"I'm hopeful we can find something before adulthood,” said McGinn. “I'd like her to have a normal life.”

Now there is hope for a cure. Researchers at Johns Hopkins say nanotechnology is showing promise and could lead to a treatment that would fix Ellie's genetic mutation and help her cells function normally. But since her disease is so rare, that research will only happen if her parents personally fund it.

Her pediatrician, Ghassan Atiyeh of Children’s Medical Associates of Northern Virginia, said if the medication is effective, the possibilities for Ellie and others are limitless.

"It could be absolutely life-altering for Ellie literally," Dr. Atiyeh said.

To fund the research project, the McGinns now need to raise $2 million over seven years.

Ellie knows her parents are working to raise money, but she doesn't know her disease is fatal. Her parents say they hope by the time she is old enough to understand that concept, it won't be the case.

"Her journey is going to make a difference,” her mother said. “I hope we find a cure for generations to come and it will all be because of her. I think it was meant to be.

“As much as I'd like to wave a wand and take it away from her, I think it was meant to happen so she can help other people."

There are three phases to the family's fundraising. The first is a goal of $61,000 by the end of November. They have raised $40,000 so far.

For more information about Ellie, you can visit her website: acureforellie.org

You can donate directly to her research project at www.KennedyKrieger.org/Ellie

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