The Courier-Mail

Aussie bub helps cure brain killer


A WORLD-FIRST cure for a rare and fatal brain disease is now saving the lives of newborns around the world thanks in part to Australian doctors.

The brain of “Baby Z” started to disintegra­te soon after her 2008 birth, with an enzyme deficiency causing a toxic buildup of sulphite in her body.

No child had ever survived the metabolic disorder Molybdenum cofactor deficiency (MoCD), which affects one in about 150,000 babies and can cause a painful death in the first few months of life.

But Monash Health neonatolog­ist Alex Veldman and the parents, who cannot be named, found a compound was being tested on animals in Germany.

After gaining approval from the Melbourne hospital’s ethics committee and the Family Court for its use in humans, Baby Z received the drug cPMP in her first month. Her sulphite levels normalised and she is now aged seven; however, she suffers severe disabiliti­es.

A study published today in The Lancet shows a daily lifelong infusion is curative in the type A variant of MoCD.

Monash University researcher Flora Wong said while Baby Z was suffering ongoing health issues, she helped save other children. “I think of her as the heroine,” she said.

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