Sydney Opera
A ferry and recreational boats pass in front of the Sydney Opera House September 28, 2014. Local media reported that the Sydney Opera House Trust has been awarded an AUD$225,000 ($200,000) grant by the U.S. philanthropic organisation, the Getty Foundation, for a study of the concrete elements of the building to develop long-term conservation strategies. It is one of 10 modern buildings around the world to be given a grant as part of a project titled "Keeping It Modern" which is aimed at conserving 20th-century architecture around the world. Picture taken September 28, 2014. Reuters

Adult patients with Pompe disease will now be granted with life-saving drug for treatment after the approval by Health Minister Sussan Ley and acknowledgement by the Australian Pompe’s Association, or APA. The government will grant $40 million funding on the extended access to MYOZYME drug for over five years under the Life Saving Drugs Programme of the Department of Health.

The health minister announced in a press release, under the Life Saving Drugs Programme, or LSDP, that patients over 18 years old with late-onset Pompe disease are now granted with expanded access to MYOZYME drug. The decision was made to expand the previous decision in February 2015 listing the drug on the LSDP for children two to 18 years old only with the infantile-onset form of the disease.

“Pompe disease is a rare and severe medical condition, affecting only a very small number of Australians every year,” Ley said, and added that the decision will improve the lives of adult patients with late-onset Pompe disease, a rare neuromuscular disorder caused by a defective gene generally characterised by progressive muscle weakness and breathing difficulty.

Covering the LSDP, the government will commit close to $40 million over five years to fund MYOZYME from September 1, 2015, according to Ley. The Pompe disease requires “a very expensive treatment, costing several hundred thousand dollars” that restricts Australians from getting regular treatment.

Under the programme, the newly diagnosed patients will undergo clinical assessment prior to accessing the treatment, followed by an annual assessment to ensure the MYOZYME treatment is clinically appropriate and effective. If the assessments show positive response from the patients, the government will continue to grant them the subsidised drug.

However, patients already taking treatment prior to the decision will be immediately eligible for ongoing treatment with LSDP-subsidised Myozyme, according to the Depart of Health. MYOZYME is a drug that contains an enzyme that naturally occurs in the body of healthy people, used to treat the glycogen storage disorder Pompe disease.

The APA president, Raymond Saich, who has been treated with MYOZYME for eight years, told PharmaDispatch, “today is a day of celebration.” Saich stated the association was grateful for the life-changing decision of the minister to make treatment available for the devastating disease.

Contact the writer at feedback@ibtimes.com.au

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