Siblings Max and Mila hope expensive drug will help change their lives

NORTHERN beaches siblings suffering from a rare disease are waiting to hear if they’ll get a life-changing drug — one of the world’s most expensive.

TWO northern beaches children suffering from a rare disease are waiting to hear if they’ll get a life-changing drug.

Max Horder, 13, and his sister Mila, 12, have a rare hereditary condition called lysosomal storage disease which is damaging their bones, affecting their growth and mobility.

It will eventually kill them.

“Their brains are not affected — they will remain alert and feel the slow degeneration of their little bodies until the disease dictates their last breath,” said mum Luba Horder, from Terrey Hills, who has launched a Facebook campaign.

Max, 13, and Mila 12, with brothers Cooper 5 and Jack 6, who are unaffected by the rare condition.

She said her children’s health had deteriorated considerably in the last 12 to 24 months.

“They used to run across grass and skip down stairs but now they struggle to maintain their balance,” she said.

“Max and Mila would like to run with their peers; they would do anything for improved endurance and a little less pain.”

Max and Mila with their mum Luba Horder.

The mother and the children’s father David, have two younger sons who are not affected by the hereditary disease.

The parents, both 46, said a government appointed group of representatives (PBAC) will this week deliberate over whether Max and Mila should receive the drug Vimizim which could halt the progression of their disease and ease their pain. The drug, however, is not a cure.

The drug could make a huge difference to the brother and sister.

Mrs Horder fears the cost of the drug which is the third most expensive in the world — $380,000 a year, per child — may affect their decision.

“If they had been diagnosed with a more common disease like leukaemia they would have immediately received the treatment they needed,” she said.

The youngsters who go to Northern Beaches Christian School were diagnosed last year, after original tests came back negative in 2009.

“Max and Mila would like to run with their peers; they would do anything for improved endurance and a little less pain.”, says mum Luba Horder.

This devastating delay in their diagnosis meant they missed the chance to be part of an earlier two-year Vimizim drug trial.

“We have considered remortgaging everything we have, but have worked out if we did we would be homeless, on the streets with four kids, in just two years,” said Mrs Horder.

A spokesman for the department of Health said the outcome would be announced on April 22.

“Mila is desperate for continued growth so that she may be able to enjoy the big girl rides at Luna Park,” Mrs Horder said.