Australian Researchers Advance Treatment for Rare Childhood Disorder KAND

KIF1A Associated Neurological Disorder, or KAND, is a rare progressive neurodegenerative condition often called a form of childhood dementia. It has no cure, but researchers at the Murdoch Children’s Research Institute in Australia are making progress on treatments.

"Their research is helping all of the kids, which is really incredible," said Luke Rosen, founder of KIF1A.org. His daughter Susannah was diagnosed with KAND at age 2. "And the folks at Murdoch, I say they're extended family to us, and they really are."

The disorder causes seizures and leads to losses in cognitive function, motor skills and vision over time. It affects each patient differently, complicating diagnosis.

Rosen said MCRI stands out for its personal approach. Researchers take time to know the families they help. "The first thing [a researcher] said to me is, 'How's Susannah? How's she doing?' And he really got to know our family, and he travels and so does their whole team... to our yearly Scientific and Family Conference, so they really get to meet all the families."

A new Fox Nation special highlights MCRI’s work and families like the Rosens, who collaborate with researchers on the debilitating disorder.

Rosen has devoted his life to advancing research and treatment options. He works closely with MCRI, where efforts already show promise.

Susannah was among the first patients to receive antisense oligonucleotide therapy. Her family reports meaningful improvements in her condition.

The treatment lacks approval in Australia so far. Rosen keeps working with doctors to broaden access, hoping families eventually will not need to travel for care.

"Their research is really translational research that helps everybody," Rosen said. "While we don't have a therapeutic yet from Australia, they're really working hard to develop it, and they're accelerating that entire process. What's special about Murdoch is they work in multimodalities, so there's gene therapy and then traditional drug development, and then they work non-sequentially, and they are just trying to throw as many shots on goal as we can for the kids."