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Aussie haemophilia patient treated with drug worth $5m a dose



All medications come at a cost — but a potential cure for a rare genetic disease comes with a seven-figure price tag.

Sydney's Royal Prince Alfred Hospital is part of a groundbreaking new trial aimed at potentially curing haemophilia B — a hereditary bleeding disorder.

Patients with haemophilia B have a mutation on the F9 gene, which leads to excessive bleeding and damage to joints thanks to the lack of a protein that helps blood to clot.

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Just 40 patients in the world will be infused with the gene therapy —with each dose the equivalent of $5 million, or more than $1000 a drop.

A safe virus carrying the F9 gene is infused into the body, delivering it to the liver cells, where doctors hope the protein is made for the rest of the patient's life.

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Professor John Rasko, Head of Cell and Molecular Therapies at RPA, described the process as a "thrilling moment in the history of gene therapy".

"We are pushing the boundaries of medical technology hoping to bring cures to individuals," he said.

"It's a massive international effort that takes time and money and ultimately the manufacturing alone costs millions and millions of dollars."

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Currently haemophilia B is kept in check with up to three injections a week. This treatment could mean one infusion a lifetime.

Brett Bardney, 52, is part of the trial.

"I feel very lucky that I have been able to participate in the program – very privileged," he said.

Monday's infusion was the culmination of more than two decades of work.

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