Without treatment our bright and loving 7 year old son Christian Dierkx will die. There is hope but this hope is being dashed by government redtape in Australia.
Christian was diagnosed with an ultra-rare genetic condition called Hunter syndrome when he was only two weeks old. The condition attacks his entire body both physically and mentally. Life expectancy for him is around 14 to 15 years of age without treatment. Michelle Dierkx , mother of Christian lost three brothers to the same condition so she unfortunately is well aware of its horrible effects.
There is no cure for the ultra-rare disease at the moment but there is a treatment called Elaprase which treats and stabilises the physical effects of the condition on the body but not the brain. Therefore the brain continues to progressively degenerate leading to death.
Shire the bio-pharmaceutical company is conducting a global clinical trial to treat the disease in the brain as part of their global phase II/III IT trial. They are willing to fully sponsor and pay for a clinical trial site in Australia.
However, the Australian health department guideline states that patients participating in a clinical trial will no longer be eligible for subsidized Elaprase which treats the body. This means Christian will no longer receive Elaprase under the government Life Saving Scheme which is a criteria for the IT trial. This is showstopper as Shire will not proceed with the trial until they are assured patients will continue to receive subsidized Elaprase.
Our son's life is at risk and without treatment he will succumb to the disease in his brain and ultimately die.
He has hope but this hope is being dashed by government redtape. We do not have time in Australia as Christian and other patients are about to miss out on this global trial. Please help us to pressure the Health Minister and the Australian Health Department to find a interpretation around this clause in the guideline which will allow the trial to proceed without delay.
