MILPERRA resident Belinda Delacour is part of a small group of people with Pompe disease awaiting the outcome of an application for the medication used in the treatment of the potentially fatal disease to be listed on the Pharmaceutical Benefits Scheme.
Australian Pompe’s Association lodged its application to the Federal Government’s panel on Wednesday, making it the fourth time it has requested funding for medication which could cost between $300,000 and $500,000 a year if not subsidised.
Myozyme is an enzyme replacement therapy used to treat the disease. It is funded by Genzyme Corporation but because of changes to the Federal Government’s Life-Saving Drugs Program, that funding could cease.
The drug’s users say it stabilises their condition, the progression of the disease is slowed and life is extended.
Ms Delacour was diagnosed with late-onset Pompe disease in 1997. The often fatal disorder disables the heart and muscles and is cause by the deficiency of the enzyme acid alpha-glucosidase.
Ms Delacour is urging for the drug to be subsidised.
“My life would be dramatically different. You find little mechanisms to cope but if I don’t have this treatment I’m going to be ventilated, at what age I don’t know,” she said.
“A simple thing like walking would be difficult.”
The submission included a letter written by International Pompe Association president Allan Muir.
“I am concerned and dismayed that a rich, developed country like Australia does not yet support its small number of Pompe’s patients, while much poorer European countries are now doing so without complaint,” he said.
“With Australia’s excellent history of Pompe’s diagnostic researching and world leading capability ... I would have expected Australia to have been a leader in its support of Pompe’s patients.”
Australian Pompe’s Association president Helen Walker said the application’s outcome could take up to six weeks to be determined.
Source: whereilive
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