“Supermarkets Don’t Sell Bones” Siblings with Lysomal Storage Disease Struggle with Funding for Medication

SIBLINGS Max and Mila Horder have a rare disease that is destroying their bones. They now face life with increasing pain and an early death because the life-changing drug which would stop their condition worsening won’t be funded as part of the government’s Pharmaceutical Benefits Scheme.

Max, 13, and Mila, 12, of Terrey Hills on Sydney’s northern beaches, have a rare hereditary condition called lysosomal storage disease which destroys the body’s bones.

The family had pinned their hopes on the drug Vimizim — which can cost up to $800,000 per person, per year — being approved on the PBS.

In a cruel twist of fate, the Northern Beaches Christian School pupils, would currently be part of a free Vimizim drug trial if their condition had not been misdiagnosed in 2009.

Mum, Luba, said the Pharmaceutical Benefits Advisory Committee’s decision would cause her children’s suffering to worsen.

“Without the drug, their bones will continue to disintegrate, their quality of life will diminish and they will continue to suffer in pain,” said Mrs Horder, 46.

The siblings’ condition will ultimately kill them.

Her daughter Mila’s response was simple.

“I just want a chance to grow tall enough to go on the big girls’ rides at Luna Park,” she said. “One day, I want to get married and have children of my own.”

Mrs Horder and husband David, 46, have two other boys who are healthy.

This week they launched a Facebook group to raise awareness of their plight, called Supermarkets Don’t Sell Bones, after one of their younger brother asked why they couldn’t just buy them new bones.

Friends have also rallied launching a petition asking Federal Health Minister Sussan Ley to intervene and grant approval for the drug for Max and Mila.

Mrs Horder said their best hope was to put pressure on the Government. However, she said the stress of it all was taking its toll on her.

“I can’t breathe most of the time, I don’t know what I’m doing,” she said. “I just want the drug for my children, that’s all I seek.”

A spokesman for the Department of Health said BioMarin, which makes the drug, had applied for listing on the separate Life Saving Drugs Program (LSDP). This application was under consideration by the Government.

Sign the petition at change.org/maxandmila.

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