Lama Al-Akhras was bracing for life in a wheelchair last week as her body struggled with a rare and fatal disease.
The 47-year-old single mother of one was diagnosed with the debilitating Pompe disease in 2012, a neuromuscular disorder that causes muscles to weaken. Since then her breathing has become more shallow and walking is difficult.
But from September 1, Ms Al-Akhras will be offered life-saving treatment by the federal government after Health Minister Sussan Ley approved the drug Myozyme to be placed on the government’s Life Saving Drugs Programme.
“I am very grateful,” Ms Al-Akhras said, crying. “I was anxious and had panic attacks and I was extremely worried about my son’s wellbeing.”
Her condition had worsened to the point that Ms Al-Akhras wasn’t sure how long she could continue walking.
“I actually did literally send an email three days ago to Muscular Dystrophy asking them to help me find a wheelchair because I didn’t know where to start,” she said.
Ms Ley will announce on Monday that the Abbott government will provide $40 million over five years to help treat the deadly disease.
“Pompe disease is a rare and severe medical condition, affecting only a very small number of Australians every year,” Ms Ley said.
“However, it is a very expensive treatment – costing several hundred thousand dollars – putting it out of reach for many Australians.”
Ms Ley said the approval for the drug had occurred after the tireless efforts by the Australian Pompe Association.
Its president, Raymond Saich, who also has the disease, said they were overwhelmed by the decision for the drug to be made so easily available.
“We are so appreciative of the minister for approving it,” Mr Saich said. “It’s going to make such a difference to so many people,” he said. “We have made such steps forward in such a short time.
“It’s the extended family who benefits from these things.”
About 30 adults in Australia have the rare disease. The drug has been listed on the Life Saving Drugs Programme for children with the infantile-onset form of the disease since 2010 and since February 2015 for children aged two to 18 years.
But adults who have the disease have had to pay up to $300,000 a year for treatment, or be offered charitable access to the drug.
“I have a family history of this,” Mr Saich said. “I lost my brother when he was 54. He was a lot older than me so he did not have the advantage of treatment. Treatment has only been available since 2006 so I was one of the first treated patients.”
Ms Al-Akhras, who has been tirelessly campaigning for help to receive the drug, said she couldn’t stop crying at the news that the drug would now be available free of charge.
“I could not believe it,” she said. “I feel like a feather now. That a load has been taken off my shoulders.”
She also hoped to take up her position as an interpreter again.
“I am so, so happy that hopefully I can pick up on work again and having a more stable future and I don’t have to be anxious about my future or anxious about my son.”