Families with 'bond deeper than blood' unite to find cure for rare disease SPG56

ABC NEWS (Australia)

The parents of a Queensland girl with a rare brain disease are joining forces with international families in a bid to find a cure. There are just 30 known cases of the disease globally. But through sheer determination they've have rallied an army of supporters and scientists to their cause.

Transcript

00:00Six-year-old Tallulah Moon has just the right mix of sweetness and sass.

00:07Do I have to make my own coffee?

00:09You need a pen.

00:10Oh, alright.

00:11Her parents feared they'd never reach this point.

00:13In 2020, their bubbly toddler, who'd been hitting all her milestones, suddenly stopped.

00:18And within about a four-week period, she couldn't hold her head up.

00:23She was on the ground, she couldn't stand, she couldn't roll over.

00:26Genome mapping revealed the cause of her decline.

00:29Hereditary spastic paraplegia type 56, which attacks the brain's nerve cells.

00:34A disease so rare, they don't know how much time they have.

00:38We can't really see what the future holds for Tallulah, because there isn't any research done on it.

00:45That's what spurred the Sunshine Coast family into action.

00:49Since their daughter's diagnosis, they've helped raise millions towards finding a cure

00:54and have connected with the handful of other affected families from around the globe.

00:59Including the Venia family from Romania, who recently came to Australia to meet them.

01:05Young Mario and Medea also have the disease.

01:08Our journey was hard and very emotional, but today we have hope.

01:13Thanks to you, thanks to the scientists.

01:15Researchers are developing a gene therapy to stop or even reverse the progressive disease.

01:21They're now on the brink of a clinical trial.

01:24It just, it both breaks my heart to see that, but at the same time go like,

01:28OK, this is the reason why we need to move at speed and why we need to do this now.

01:33As her parents continue to fight for a cure, Tallulah joined her big brother at school this year.

01:39In the first week, the feedback we got from the teachers was that,

01:43no, look, she wasn't having any trouble fitting in.

01:46She was more like Taylor Swift and they had to sort of push the fans away from Tallulah to give her space.

01:52Or that one. Or this one. I'll put it here.

01:56.

Category

Transcript

Recommended